Winter 2009 - The Canadian MPS Society

Highlights
in this issue:
Medical Advisory Board
Member Profile: Dr. Julian
Raiman—page 6
Parents & patients meet in
Toronto—page 11
Taiwan hosts the AsiaPacific MPS conference &
symposium on rare
diseases—page 20
Toronto Scotiabank Waterfront Marathon—page 24
Mark your calendars for
the Society’s National
Family Conference:
July 23-25, 2010—page 14
Winter 2009
Happy Holidays from the Canadian MPS Society!
InThis Issue
Director Reports/Welcome/Membership Renewal
4
MAB Member Profile—Dr. Julian Raiman
6
Donations
7
Memorials /Birthday Greetings
8
Correspondence/Family News
10
Toronto Regional Meeting
11
Advocacy
12
Upcoming Conferences & Events
14
Research & Clinical Trials
16
2009 JAPAC Educational Forum in Taiwan
20
In the News
22
Fundraising—Toronto Waterfront Marathon
24
Fundraising—A Night of Enchantment
26
Fundraising—Walk with Nature
27
Fundraising—Hope Shines Bright Gala
28
Fundraising
29
Society Merchandise
32
Society Forms
33
Family Assistance Program Funding Application
34
Society Publications
35
Season’s Greetings/Holiday Art Contest winner
36
THE CANADIAN SOCIETY FOR MUCOPOLYSACCHARIDE
& RELATED DISEASES INC.
PO Box 30034, RPO Parkgate
North Vancouver, BC V7H 2Y8
Tel (604) 924-5130/1-800-667-1846
Fax (604) 924-5131
kirsten@mpssociety.ca/www.mpssociety.ca
Charity #12903 0409 RR0001
The Canadian MPS Society’s Mission Statement:
The Canadian MPS Society is committed to:

Providing support to families affected with MPS and
related diseases.

Educating medical professionals and the general public about MPS and related diseases.

Raising funds for research so that one day there will
be cures for all types of MPS and related diseases.
What are MPS & Related Diseases?
Lysosomal storage diseases (LSDs) are rare, progressive genetic
disorders. Often, these diseases are inherited from parents
who have no idea they carry the responsible recessive genes.
People born with MPS and related lysosomal storage diseases
cannot produce certain enzymes necessary for breaking down
and recycling cells. Consequently, these cells store throughout the bodies of those with LSDs, in areas including their
hearts, respiratory systems, bones, joints, and central nervous
systems.
Babies may show no signs of disease, but as more cells become
damaged and storage increases, symptoms begin to appear.
For more detailed information about individual diseases,
treatments and research, please visit our website at
www.mpssociety.ca or order one of our disease-specific booklets from the order form on page 35.
Support for families.
Research for a cure.
Smiling faces on the front cover:
Nathaniel Phaneuf (MPS II) and his sister Chelsea choosing their Christmas tree.
“Nobody, but nobody,
can make it out here
alone”
-Maya Angelou
Page 2
Do you have family news, photos,
fundraising news or an interesting
article to share with
the Connection’s readers?
Newsletter submission deadlines:
Please submit by email, if possible,
to kirsten@mpssociety.ca or
jill@mpssociety.ca
Fall Issue:
Spring Issue:
Summer Issue:
Winter Issue:
February 1st
May 1st
August 1st
November 1st
The Connection
Board of Directors and Medical Advisory Board
Executive Director:
Kirsten Harkins
PO Box 30034, RPO Parkgate
North Vancouver, BC V7H 2Y8
MPS I parent
(604) 924-5130
kirsten@mpssociety.ca
Jill Ley, Executive Assistant
Mary Nelis, Quebec Representative Robin Casey
Bedford, QC
MPS I parent
(450) 248-7376
mary.nelis@sympatico.ca
Joe Clarke
Bernie Geiss
North Vancouver, BC
Professional
(604) 924-3451
bgeiss@shaw.ca
jill@mpssociety.ca
Todd Harkins
Board of Directors: Judy Byrne, Chair
Guelph, ON
MPS I parent
(519) 836-5949
judy@mpssociety.ca
Carrie Nimmo, Vice—Chair
Vancouver, BC
MPS I aunt
(604) 255-5456
carrie@mpssociety.ca
Jean Linden, Secretary
Prince George, BC
MPS III parent
(250) 564-3698
jean@mpssociety.ca
Brenda MacLean, Treasurer
Professional
(604) 980-7556
goodtogreat758@hotmail.com
Barbara Boland, Newfoundland
Representative
St. John’s, NFLD
MPS III parent
(709) 753-7874
barbara@mpssociety.ca
M.D., M.S.C.
Alberta Children’s Hospital
Calgary, AB
North Vancouver, BC
MPS I parent
(604) 929-8969
todd@mpssociety.ca
Aubrey Hawton
Moonstone, ON
MPS III parent
(705) 835-5288
aubrey@mpssociety.ca
M.D., Ph.D.
Hospital for Sick Children
Toronto, ON
Mark Ludman
M.D., F.R.C.P.C., F.C.C.M.G.
IWK Health Centre
Halifax, NS
Serge Melancon
M.D.
Montreal Children’s Hospital
Montreal, QC
Cheryl Rockman-Greenberg
M.D., C.M., F.R.C.P.
Winnipeg Children’s Hospital
Winnipeg, MN
Randall Linton
Toronto, ON
MPS III parent
(416) 223-0078
randall@interiorcare.com
Julian Raiman
Deb Purcell
Tony Rupar
Vancouver, BC
MPS II Parent
(604) 222-2767
dcehak@telus.net
M.D.
CPRI
London, ON
M.D.
Hospital for Sick Children
Toronto, ON
Sylvia Stockler
Medical Advisory Board:
M.D.
BC Children’s Hospital
Vancouver, BC
Lorne Clarke, Chair
M.D,C.M.,F.R.C.P.C.,F.C.C.M.G.
University of British Columbia
Vancouver, BC
Accountant: Quantum Accounting, Vancouver, BC
Canadian MPS Society Bank Account: TD Canada Trust
Winter 2009
Page 3
Chair’s Report
Season’s Greetings from the Board
of the Canadian MPS Society. May
the season bring you peace of mind
and peace of spirit.
The board is now starting
to focus on our upcoming family
conference in Alliston, Ontario.
Please make sure to set aside July
23 to 25, 2010 and join us and
other MPS families for an informative and enjoyable weekend.
On October 23, 2009, I had the pleasure of attending the Emma-Rose Gala in honour of Emma-Rose GrenonLeMaitre and her family. Lara Pietrolungo, a long time
friend of the LeMaitre’s and a nurse at Toronto’s Sick Kids,
took it upon herself to put this gala together. It was a truly
elegant evening, and all proceeds were directed to the Hospital for Sick Children in Toronto, and the family assistance
programme at our Society. Many thanks to Lara and the
LeMaitres for their continued support.
Les membres du conseil d’administration de la Société MPS
vous souhaitent leurs meilleurs voeux pour la saison des fêtes!
Que la saison des fêtes vous apporte la paix et la tranquillité
d’esprit.
Le Conseil d’administration commence maintenant
sa focalisation en vue du prochain congrès de la famille, qui
aura lieu du 23 au 25 juillet 2010, à Alliston, Ontario. Prière
d’inscrire ces dates à votre calendrier afin de vous joindre à
nous et aux autres familles MPS, et ainsi profiter d’une fin de
semaine agréable et informative.
Le 23 octobre 2009, j’ai eu le plaisir d’assister au
“Gala Emma Rose” dédié à rendre hommage à Emma Rose
Grenon-LeMaitre et sa famille. Lara Pietrolungo qui est une
amie de longue date de la famille Lemaitre et aussi une infirmière à l’hôpital Sick Kids de Toronto, a pris l’initiative d’organiser ce gala. Ce fut une soirée élégante, et toutes les recettes ont été réparties et versées à l’hôpital Sick Kids de Toronto
et à notre Société, pour notre programme d’aide aux familles.
Merci beaucoup à Lara et à la famille LeMaitre pour leur collaboration soutenue.
All the best to everyone and I wish you a happy and healthy
New Year!
Judy Byrne
Tous mes meilleurs voeux et bonne et heureuse année, en
santé.
Judy Byrne
Welcome New Members:
Jeremy Wighton
Jeremy is the father of Abigail (MPS IH)
Matthew Santos
Matthew is a new adult member with MPS IVB
Candice Riddell & Mike Taylor
Very best wishes for a wonderful Holiday
Season and a Happy Year in 2010.
-Jill Ley and family
Candice & Mike are the parents of Kobe (MPS II)
2010 Membership Renewals
Recently, you will have received an email reminder to renew your membership for 2010 online at
www.mpssociety.ca. If we do not have your email address, you’ll receive a hard copy form in January.
Regardless of the time of year we receive your renewal, membership is for one calendar year only.
Your renewed membership will be valid through December 31, 2010.
When you renew online, you will receive a PDF income tax receipt, your membership will be updated
automatically, and the Society will save on printing and postage costs.
Please first login to your account, then renew your membership and update your profile so that we have your
consent to include your contact information in our membership directory and to reprint your child(ren)’s
photos, birthdates and/or memorial dates. Please note that your profile must be updated annually.
You can find a Membership Form on page 33 if you prefer renewing the ‘old-fashioned’ way!
Thank you for your continued support!
Page 4
The Connection
Executive Director’s Report
Happy Holidays, everyone!
It’s that time of year again when love, hope, joy, and generosity are
most plentiful. As Oprah put it in her holiday issue of “O” magazine, “Let’s
keep that giving thing going all year long!” Wouldn’t it be wonderful if we could
do just that? For now, we’re hoping that our members and supporters will give
thanks over the holidays for the healthy children in their lives and give generously to those children who are not as fortunate. Please contribute if you can to
our Annual Fund Campaign and so that we can continue to support children,
adults, and entire families affected by MPS and related diseases in 2010.
In the past few months, we have been blessed by many acts of generosity and kindness—read about several fundraising events on pages 24—29. Thanks
to all involved for raising awareness and funds—none of us can do this alone!
One of my favourite events during the fall was our Toronto regional
meeting (see page 11), as it’s so invigorating to actually see in person some of the
members I correspond with throughout the year. Seeing old friends and meeting new ones made me even more excited about our national family conference,
to be held this summer at the Nottawasaga Inn near Toronto. Please join us
there! More details on registration will follow in our spring newsletter, and conference details will be posted on our website as planning continues.
I was very fortunate to have the opportunity to travel to Asia in October (see page 20) and meet many people affected by the same issues we deal with
here in Canada: Dedicated doctors, brilliant researchers, caring parents, amazing
Happy Holidays from the Harkins family:
children—all from very different cultures but all with the same challenges and
goals as us. It was inspiring to imagine what we can do as a global team. As
Todd, Kirsten, Jonas (8), Jansen (12)
Helen Keller said, “Alone we can do so little; together, we can do so much.”
& Nicklas (14)
Wishing all of you peace and health throughout the coming year,
Kirsten Harkins
PLEASE SUPPORT OUR 2009 ANNUAL FUND CAMPAIGN!
Enclosed with this issue of the Connection, you’ll find a letter from Candice
Riddell (mother of Kobe—MPS II) describing how important the Society has
been to her throughout her family’s journey with MPS. While we recognize
2009 has been a tough financial year for many Canadians, it has also been a
challenging fundraising year for the Society. We rely on private donations in
order to be able to continue to provide families like Candice’s—and yours—with
the education, support, and hope necessary for a brighter future.
We are asking our members to contribute—and encourage others to contribute
as well—to the Society’s Annual Fund Campaign this holiday
season. Give a gift to support those who are affected by MPS.
Thank you for your support!
Winter 2009
Page 5
Medical Advisory Board Member Profile
Dr. Julian Raiman
WHY DID YOU BECOME A DOCTOR?
I wanted to be a Paediatrician, rather than a doctor per se. I
would like to think it is because of a Cary Grant film I saw
when he played a Paediatrician, but expect my medical family
has something to do with it!
WHAT LED YOU TO BECOME INVOLVED IN MPS
DISEASES?
After Paediatric training in England I came and did fellowship
at Sick Kids under Joe Clarke, who taught me a lot about MPS
diseases. He and the families at the Hospital spurred my
interest.
WHAT INSPIRES YOU?
The parents and children I look after.
TELL US A LITTLE BIT ABOUT YOUR FAMILY:
I am married with two daughters aged nine and eleven. I am
the youngest of six, with four sisters – so I know where I stand
in the pecking order (my close family might disagree though!).
PERSONAL INTERESTS/HOBBIES:
Spending time with my family and an unhealthy interest in
West Ham United (a struggling English soccer team).
WHAT WOULD WE BE SURPIRSED TO LEARN
ABOUT YOU?
I was a premature baby born at 32 weeks (quite early back
them!) and I directed a play at the Edinburgh Fringe Festival
while at medical school – which won the award for the worst
show there in 1989.
Dr. Raiman speaking at our recent parent & patient
meeting in Toronto about a cognition study he will
be facilitating at Sick Kids. For more information
on this study, please see page 19.
$$ Do you know about the Canadian MPS Society’s Family Assistance Program? $$
Grants of up to $1,500.00 are available to individuals or families affected by MPS or a related
disease, and can be used to offset emergency or non-emergency costs associated with care and
management. Do you need a special piece of adaptive equipment that your insurance will not
cover, or will only cover a portion of? Do you need home renovations to accommodate a wheelchair? View our FAP guidelines at www.mpssociety.ca under “support” and call with any questions.
An application form is available for download on the website or on page 34 of the Connection.
We also have Family Assistance Program funds specifically allocated for respite care.
If you need a break from caring for your child(ren), we would like to help! Call for details.
Page 6
The Connection
Donations
Todd Campbell
Linda Rooney
Nuha Alabssy
Brenda Casselman
Lisa Bernais
Biomarin Pharmaceutical Inc. (Canada) Donna Rooney
Harmony Rooney
Shirley & Barry Bowser
Tracy & Michael Davison
Pierre Chayer & Christiane Parent
Dennis Roth
Coiffure De L'Estrie
Jennifer Wren
Ruth Goff
Terry Turner
Norman Goyette
Bob Klages
Margaret & Gerald Greig
Jill Reekie
Dorothy Harkins
Steve Pendleton
Frank & Verle Kaweski
Dave Pal
Allan & Karen Knight
Maxwell Alexander Settari Fund-Calgary Patti Haney
Brittany Rooney
Foundation
Lorene Taylor
William & Louise Parker
Julie Douglas-Vail
Rob & Lisa Patriquin
Mike Kearney
Genevieve Poulin
Thomas & Verna Wilcox
Candice Riddell & Mike Taylor
Nora Delmas
Vanessa Rooney
Marion Lee
Ms. Esther Sherbanow
Marilyn Moore
Shire Human Genetic Therapies
Valerie Cribbs
United Way Burnaby
Steve Ashley
Robert & Shirley Young
Rosemary Palmer
MPS II Research & Time for a Cure Angela Pyatt
Jeffrey Robins
Donors
Jen Hanbidge
Tanja Balic
Diane Haines
Edward & Dorothy Keen
Sue Cramp
Henry Stulen
Jill Calvert-Goetz
Mary Loveday-Wight
Toronto Waterfront Marathon
Erica Rooney
Donors
Dana Rooney
Bill & Jennifer Pearson
Dave Stewart
Dave & Sue Lambe
Lindy Balasuriya
David & Sonya Glass
Brad Forrest
Catherine & Jim Fowler
Deb Ferguson
Robert & Cara-Lee Moore
Randy Osborn
Barb Murray
Paul Woolner
Christine Graham
Jill Robinson
Ruth Henning
Shelley Cornfield
Nancy Fry
Donna Spetko
Ron McNabb
Kip Barber
Paul Amador
Brad Toole
Joan Keeling
Barb Collins
Donations
Winter 2009
Danette Welford
Terry McKauge
Jim Brown
Paul Lackey
Cove Cliff Elementary School
Jeans Day Donors
Shairoz Alibhai
Christy Andersen
Deanna Barlow
Janet Brock
Christene Buchanan
Theresa Lake & Christopher Burt
Nina Chen
Wayne Davidson
Fiona Douglas-Crampton & Dan Arrigo
Bob & Chris Eldon
Jordyn Eyton
Johan & Anna Fourie
Kirsten & Robert Hannah
Nicole Huth
Nora Koller
Ellen Leroux
Ken Huckell & Joanne Maltby
Matreya Monro
Steve Weller & Catherine Moore
Maureen Palmer
Nora Pickett
Colleen Quee-Newell
Gil & Jessica Rosenfeld
Karen & Tim Saxvik
Mike & Diana Smedstad
Christine Tevlin
Lori Weston
Emma-Rose Fund Gala
Randall & Elisabeth Linton
Hafiza Chiragdin
Michele LaPerriere
Avedis Oudabachian
J.T. Watson
Marie France Mistele
Jessica Peterson
Brenda Deseure-Casteels
Mary Monty
Page 7
Donations
Emma-Rose Fund Gala
Walk with Nature Donors
Donors (cont’d)
Various cash donations
Martha Monty
Jonathan Monty
John Chesney
Kim Logan
Verna Maurice
Bonnie Smith
Gaetan Beaulieu
Nancy Richard
Ontario Fluid Power Ltd.
Boulevard Studios Inc.
Canadian Building Materials
York Roofing
Vera Gueorguieva
Gabriel Sadaka
Suzanne Lam Tim Woo
Grenville Pinto
Denis Geary
Rachel Hostetler
Dawn Irving
Donna Langlois
Gail Ribaux
Jennifer Luce
Lyne Villeneuve
Karen Christiansen
Pamela Bertram
Daniella Bernstein
France Gagne
Metz Canada Ltd.
Bell, Temple
Great Lake Walk Donors
Ken & Helga Irving
In memory of Robert Johnson
Peggy Johnson
Esther Sherbanow
Leslie & Kathleen Staley
James & Dorothy Reid
Marion Lamb
Peter & Karen McDonald
Mrs. Alva Brennan
Mrs. Doris Douglas & Mrs. Carol Boyce
William & Betty Porterfield
Daniel & Gertrude Novak
Carolyn Miller
Shirley Staley
Donald & Charlotte Buckle
Margaret Belcher
Richard Belcher
Linda & Mel Tatarka
John & Elda Bannon
David & Henrica Maguire
Stan & Louise Airdrie
Robert & Patricia Johnson
Ron & Cheryl Carriere
Dave & Sandy Klein
Tammy & Alan Jones
Modern Heating Brantford Ltd.
In memory of Giuseppina Sardo
Vito Schiavone
Carmelo & Luisa Vignanello
Vincenzo & Nilla Distefano
In memory of Maria Russano
Rob & Lori Di Ilio
In memory of Margaret Simpson
Michelle Campbell-Wood
In memory of Philip Bojan
Dr. Joe Clarke
In Honour of Matthew Di Ilio’s 19th
Birthday
Rob & Lori Di Ilio
Remembering our Children:
Bill Baillie: March 10, 1969 – January 8, 2000 — MPS II
Abdullah Alglaieny: September 14, 1987 – January 15, 2008 – Fucosidosis
Scott Haines: November 21, 1963 – January 27, 1991 — MPS III
Jenna-Anne Woodworth: September 20, 1983 – March 11, 1989 — MPS
Lindsey Olsen: April 25, 1983 – March 19, 2002 — MPS III B
Jason Furoy: August 30, 1979 – March 30, 2003 — MPS I S
Jamie Borys: July 30, 1993 – February 18, 2007 — MPS III A
Page 88
The Connection
Birthday Wishes
Happy Birthday !
JANUARY
Kobe Taylor – January 12, 2005 – MPS II (Hunter)
Reece Polowski- January 14, 2003- MPS I-H (Hurler)
Peter Bauerle – January 17, 1970 – MPS
Gregory Alan Lipton – January 21, 1989 – Mucolipidosis Type IV
Raza Amin- January 25, 1999- MPS III B (Sanfilippo B)
FEBRUARY
Sheena Hodder – February 4, 1985 – MPS IIIA (Sanfilippo A)
Jean Luc Amirault - February 23, 2000 – MPS I-H (Hurler)
MARCH
Trey Purcell- March 1, 2004- MPS II (Hunter)
Colin Collins – March 9, 1975 – MPS III A (Sanfilippo A)
Andrew Lanese - March 10, 1997 – MPS II (Hunter)
Sophie Marie Hamza – March 14th, 2008 – MPS IIIB (Sanfilippo B)
McKay (Mac) Stiles – March 21, 2004 – MPS I-H (Hurler)
Elisa Linton – March 31, 1994 – MPS III B (Sanfilippo B)
We cannot print your child’s name on our Birthday list without your written consent to do so. If you would like your child’s name to
appear on the Birthday list, please indicate on the current year’s Society membership form. We apologize if any names are inadvertently
missed. Just give us a call and we will make sure your child’s name appears in the next edition of The Connection. Happy Birthday!
Winter 2009
Page 9
Correspondence/Family News
Kobe Taylor
The Johnson Family (MPS I-H)
(MPS II)
Brantford, ON
Mississauga, ON
Thanks so much for your card. I’m so glad that most people
contributed to MPS in Bob’s memory. I’m so glad I thought
I've been meaning to call
of going this route as Bob was always so good about taking me
you to update you on how
to see the boys even though it was a 2 -3 hour trip and visits
treatment has been going
short as they didn’t even know we were there. Sometimes we
for Kobe.
made weekend of it so Melody could enjoy a swim and fun at
This Tuesday will
a hotel and I could see Kevin and Scott both days.
be his sixth treatment
Even though he (Bob) went into hospital December 7, 2008
session and its been wonand never got back home we both knew this would be the
derful. He has had no bad
ending and yes, I really do miss him terribly and now that I
reactions and seems to be responding to the medication well.
am finally wrapping up the paper work, I find it hard to get
I am very pleased and so is Mike.
myself going each day after spending 9 months going to either
Dr. Raiman came to see us on Monday and was very
hospital or nursing home every day.
happy with how well Kobe is doing. Dr. Raiman said that it
Thanks again for your help and being there, and
should take 8 to 12 weeks before seeing any kind of change.
when I need to send a donation for someone else I will send
Mike and I think some things have started to change already.
it to MPS. Your sympathy cards are lovely.
We go every Monday starting at 8am.
Sometimes his
Sincerely,
infusion doesn't
Peggy Johnson
even start until
10am, so it’s a
(Peggy’s husband Bob passed away in September 2009. Her sons
very long day for
Kevin and Scott had MPS III . Her daughter Melody is unaffected)
Kobe and us but
he is such a good
boy. We try and
keep him entertained for as long
as possible. The
first few weeks
Kobe would sleep for two hours and now he stays awake but
is very drowsy.
I just want to thank you for all the help you gave us.
I could not have gone through this without your support.
I really believe in my heart that if you were not around to help
Kobe he wouldn't be having his treatment.
I feel like you were the only one who understood the frustration and heartache I was going through as a mother.
I could thank you a billion trillion times but it will never express the gratitude I have for you and how much you mean to
us.
Well I've talked
your ear off.
Hope everything
is well for you
and your family.
Take care talk to
you soon.
Candice Riddell
Page 10
Melissa Bilodeau (MPS IVA) with best friend ‘Papuk’
The Connection
Toronto Regional Meeting
An Evening for Parents and Adult
Patients affected by MPS Diseases
On November 10th, close to forty people affected with or by
MPS gathered in Toronto for a regional meeting hosted by
the Canadian MPS Society. We were delighted to have parents of children affected by MPS I, MPS II, MPS III, MPS IV
and MPS VI in attendance, as well as adults affected with
MPS IV and VI—a few of whom I had never met before but
got to know over the course of the evening! I was also pleased
to see our charming friend David Mackay (Mannosidosis) and
his parents at the meeting, and to hear about the award David
won recently for his service to the Region of Durham through
his volunteer work at the Fairview Lodge (see photo!).
Kudos must go out to the amazing Margaret
Mackrell, Ontario’s LSD coordinator at Sick Kids and nurse
extraordinaire, who singlehandedly catered our dinner. The paninis were
delicious! We also must
thank Dr. Julian Raiman,
who took time from his
very busy schedule to
speak to our group about
the MorCAP natural history study (read more
about it on page 18) and a
“Caterer” Margaret Mackrell longitudinal cognition
& Iffat Amin enjoy a chat study for which he will be
recruiting patients in the
very near future (read more about that study on page 19).
Thanks, too, to Sick Kids social worker Karen Sappleton, who
described a new grant program being developed for families
with kids with MPS through the hospital’s Patients Amenities
Program. This program will be available for families whose
children are treated at Sick Kids and will be funded through
money raised at the “Night of Enchantment” event, which
was inspired by Emma Rose Grenon-LeMaitre (MPS I) and
took place October 23rd in Toronto (see page 26). For more
Winter 2009
information on this program,
please email Karen at
karen.sappleton@sickkids.ca.
Lara Pietrolungo, a
Sick Kids nurse who enthusiastically organized the fundraiser, presented me with a
cheque for $5,000 toward our
Family Assistance Program
(FAP) just before the meeting.
Kirsten with David
As the Sick
Mackay
(Mannosidosis)
Kids fund
will only
be available to those families who use that
particular hospital, we appreciate the donation to our FAP, which of course is available to all our member families. Thanks so
much for your energetic support, Lara!
The evening concluded with an
update on Ontario’s Rare Disease Program
by Dr. Durhane Wong-Reiger, president of
Canadian Organization of Rare Disorders
(CORD). Durhane also filled us in on
Canadian Institute of Health Research’s (CIHR’s) plan to
launch a grant program specifically for rare diseases—an exciting development
that we’ll keep you
updated on!
Thanks to Durhane for joining
us, and also to
Sick Kids Medical
Genetics nurses
Michelle Mecija
and Dawn Cordeiro for being
Durhane Wong-Reiger speaking
such strong supporters of the families they serve.
Thanks to all who made the effort to attend the Toronto regional meeting. It was so wonderful to be together
with many of our Toronto-are members, and I look forward
to seeing you again this summer at our National Family
Conference!
Kirsten
Matthew Santos
(MPS IVB), his girlfriend, & Verica
Gacic have a visit
Page 11
Advocacy
ERT Funding for MPS I
All individuals requiring Aldurazyme enzyme replacement
therapy in Canada are being treated, although the funding
agreement currently in place will expire soon and will have to
be extended or renegotiated. We do not anticipate any interruptions in treatment for patients receiving Aldurazyme.
ERT Funding for MPS II
Since Elaprase enzyme replacement therapy for MPS II was
not recommended for funding by the Common Drug Review’s (CDR’S) committee, it has been up to provincial, territorial, and federal funders to either follow that recommendation, or decide to fund regardless. Children in BC, Alberta,
and Quebec area now being treated (albeit in Quebec, the
funding is through private insurance), and Ontario’s new
Drugs for Rare Diseases program is funding patients in Ontario on a case-by-case basis. Funding is not available in Sas-
U.S. and European Rare Disease Organizations Sign Strategic Alliance
Danbury, CT-----The leading advocacy groups for Americans
and Europeans with rare diseases—the National Organization
for Rare Disorders (NORD) and the European Organization
for Rare Diseases (EURORDIS)—have signed a Memorandum
of Understanding to join forces on several key strategic initiatives on behalf of rare disease patients and their families.
The intent is to increase global awareness, promote
research and the development of new treatments, and provide
advocacy for more compassionate public policies.
"Collaboration between our two organizations will
make both stronger and more effective in representing our
constituents," said Peter L. Saltonstall, President and CEO of
NORD. "Since patients with rare diseases may be located
anywhere in the world, international collaboration to support
and advance their needs is extremely important. This strategic
partnership will help us address issues and challenges that are
essentially the same on both sides of the ocean."
Yann Le Cam, Chief Executive Officer of EURORDIS, noted that "EURORDIS and NORD are in a
unique position to promote solidarity between Europe and
the US and help bridge the gap between those regions of the
world that have progressed more than others in their strategies
to address the needs of people living with rare diseases. We
can be key facilitators to promote the common interest of
people with rare diseases and to empower patient advocates all
over the world."
As part of their strategic partnership, EURORDIS
and NORD will:

Co-organize an annual global Rare Disease Day

Establish common positions on key advocacy priorities
Page 12
katchewan or New Brunswick, and we are working to address
that situation. Thank you to those of you who have participated in our electronic letter-writing campaign: stay tuned for
more ways you can help secure funding for ALL individuals
with MPS II needing treatment.
Parents of Canadian children affected with MPS II,
and adults with MPS II, should discuss Elaprase with their
children’s, or their own, physicians. Shire has established a
1-800# for families interested in exploring potential funding
options available through private insurance. For more information on Elaprase or the Shire Reimbursement Support
Program, please contact Kirsten at the Society’s head office.
And What about MPS VI?
A small number of Canadians are receiving ERT to treat their
MPS VI. Funding decisions for Naglazyme will continue to be
made on a case-by-case basis.




Play a pivotal role in the International Conferences for
Rare Diseases and Orphan Drugs (ICORD) to help
expand the rare disease movement
Collaborate in the development of international web
media and social networking information and communication services
Implement an international mentoring project for rare
disease patient organizations
Coordinate more closely activities to enhance relations
with the pharmaceutical and biotechnology industry
EURORDIS and NORD will kick off their collaboration with
a joint public policy advocacy blog and shared online communities.
This collaboration among patient advocacy leaders
mirrors other recent developments. The U.S. Food and Drug
Administration (FDA) and its counterpart in Europe, the
European Medicines Agency (EMEA), have announced a collaborative effort to ensure appropriate conduct of clinical trials. The two agencies have also adopted a common application for "orphan" products—products being developed as treatments for rare diseases.
A disease is considered rare in the U.S. if it affects
fewer than 200,000 Americans. Almost 30 million or nearly
one in 10 Americans have rare diseases. In the European
Union, a disease is considered rare if it affects fewer than one
in 2,000 people. There are 30 million Europeans with rare
diseases.
NORD, established in 1983, and EURORDIS,
founded in 1997, are both non-governmental, patient-driven
alliances of patient organizations and individuals dedicated to
improving the lives of people with rare diseases. The NORD
website is at www.rarediseases.org The EURORDIS website is
at www.eurordis.org
The Connection
What you should know before submitting your tax returns
Federal Tax Initiatives provide support to
disabled individuals and their families
CDB payment will be included in the Canada Child Tax
Benefit payment. When filing a tax return, families will continue to be able to claim the disability amount and corresponding supplement for qualifying children.
Disability Tax Credit
For more information on the disability amount, visit CRA’s
People with disabilities page or call T.I.P.S. at 1-800-267-6999.
The Disability Tax Credit is a non-refundable tax credit used
to reduce income tax payable for eligible individuals. To be
eligible, you must meet all three of the following conditions:

The CDB is based on family net income and provides up to a
maximum of $199.58 per child each month to families with
eligible children. For more information about the CDB, visit
You must have a severe impairment in physical or men- CRA’s Child and Family Benefits page or call 1-800-387-1193.
tal functions.

Your impairment must be prolonged, which means it
Child care expenses
has lasted or is expected to last for a continuous period
You or your spouse or common-law partner may have paid
of at least 12 months.
someone to look after your child who, at some time in 2009,

A qualified practitioner certifies that your impairment
was under 16 years of age or had an impairment in mental or
is severe and prolonged and completes Form T2201,
physical functions. Generally, the spouse or common-law partDisability Tax Credit Certificate, detailing the effects
ner with the lower net income can claim these expenses, but
of the impairment applicable to the basic activities of
only if the expenses were paid so one of you could earn indaily living.
come, go to school, or do research in 2009.

If you are eligible for this credit but are unable to use
all or part of it because you have no taxable income,
More Medical and Disability-Related Information
you can transfer it to your spouse, common-law partner, or other supporting person.
The CRA has a guide about disability-related information
which is meant for people with medical expenses or impairA supporting person may be able to claim all or part of a dements in mental or physical functions and anyone supporting
pendant's Disability Tax Credit providing that both the supthese people. It gives information on eligible medical exporting person and the dependent were residents of Canada
penses, services provided for people with impairments in menduring the tax year.
tal or physical functions, as well as other information on income tax, excise tax, customs, and the goods and services
How do I find out if I’m eligible?
tax/harmonized sales tax (GST/HST).
Find out if you may be eligible for the Disability Tax Credit by You can view and download this guide at:
answering questions from a self-assessment questionnaire
http://www.cra-arc.gc.ca/E/pub/tg/rc4064/rc4064-08e.pdf
available at:
http://www.taxspecialistgroup.ca/disability/content/eligible_
en.htm
Need help filing your taxes?
This questionnaire does not replace the form itself. It is provided to help you assess whether you may be eligible for the
Disability Tax Credit.
Child Disability Benefit
Families with children who qualify for the disability amount
may be eligible to receive the Child Disability Benefit (CDB).
A child qualifies for the disability amount when you have approved Form T2201, Disability Tax Credit Certificate, for that
child. The CDB will be calculated automatically for the current and the two previous benefit years for children who qualify and are under 18 years of age. If you want to get the CDB
beyond these benefit years, send a letter to your tax office. The
Winter 2009
Try the CRA Community Volunteer Income Tax Program
If you need help to complete your tax return, your income is
low, and your tax situation is simple, CRA volunteers are specially trained to help you. For more information about this
free program, visit the CRA Community Volunteer Income
Tax Program page or call us at 1-800-959-8281.
In-person appointments
If CRA cannot resolve your enquiry by telephone, you can
arrange to meet with an agent at a tax services office by calling 1-800-959-8281 or if you are using a TTY, 1-800-665-0354.
Our premises include ramps and other aids to make entry
easier and to prevent undue physical demands.
Page 13
Upcoming Conferences & Events
Upcoming conferences & events:
December 17-18, 2009: National MPS Society’s
National Family Conference. Disney World, Orlando,
Florida. www.mpssociety.org
February 10-12, 2010: The 2010 “WORLD” Symposium, Miami, Florida. Hosted by the Lysosomal Disease Network. www.lysosomaldiseasenetwork.org
May 8, 2010: Tacos for Trey. North Vancouver, BC.
Visit www.treypurcell.com for more information.
May 15, 2010: International MPS Awareness Day.
Watch for news in our spring issue of the Connection.
May 15, 2010: The 8th MPS CUP Fantasy Hockey
Game & Gala. Vancouver, BC. www.mpscup.ca
MPS Registries & Websites:
The MPS I Registry: A resource available for your
physician or health care professional that is dedicated to improving the understanding of MPS I. With the MPS I Registry , your physician can access your data and compare it to
aggregate data from around the world. Ask your physician to
call 1-800-745-4447, ext. 17021 for more information.
The MPS II Registry: The Hunter Outcome Study
(HOS) is a new registry dedicated to patients with MPS II
(Hunter Syndrome). HOS is designed to support the gathering, analysis, reporting and sharing of data from around the
world about Hunter syndrome. More information about
Elaprase and MPS II (Hunter Syndrome) is available at
www.elaprase.com.
June 23-26, 2010: The 11th International Symposium on MPS & Related Diseases—Translating
Research into Clinical Reality. Adelaide, South Australia. See the ad on page 15 for more information,
and visit www.mps2010.com.au for updates.
MPS IV Registry: Information about MPS IV can be found
at www.morquio.com. Also available at this website is the
Morquio registry—once registered, it is recommended that
updates be made at least yearly, in order to ensure natural
history data is captured to provide evidence critical for developments of treatments for MPS IV.
Please join us at our 2010 National
Family Conference!
MPS Websites:
Where: The Nottawasaga Inn
Alliston, Ontario (about 1 hour from Toronto)
When: July23—25, 2010
Why: To meet with other families and
individuals affected by MPS, and
renowned professionals….
Share, learn and laugh together!
Travel bursaries will be available to
Society members. For more
information, visit www.mpssociety.ca
or email info@mpssociety.ca.
Page 14
www.MPSIdisease.com. A Genzyme-developed website with
information and resources on MPS I.
www.Aldurazyme.com. A Genzyme-developed site, providing parents and patients with information on Aldurazyme.
www.lysosomallearning.com. A Genzyme-supported online
resource relating to lysosomal storage disorders (LSDs).
www.MPSII.com. A Shire-developed website, providing information about MPS II (Hunter Syndrome).
www.MPSVI.com. A BioMarin-developed website, with
information relating to MPS VI.
www.MPSVI.net. Register for free on this site to connect
with your MPS VI community.
www.naglazyme.com. This new Biomarin-sponsored website
is now live. You will find expanded content about MPS VI, its
diagnosis and treatment with NAGLAZYME® (galsulfase)
enzyme replacement therapy, and much more.
www.goldinfo.org. Global Organisation for Lysosomal Diseases’ website, offering LSD information and resources, as
well as videotaped presentations from conferences.
Links to international MPS society websites can be found
on our website: www.mpssociety.ca.
The Connection
2010 International MPS Symposium
The 2010 Organizing Committee invites you to join us in Adelaide for the 11th International MPS Symposium.
Our wish for this meeting is for it to be a positive learning experience for all who attend and as such we wish to
maintain the high scientific standards of previous meetings, by planning sessions that will be both informative
about issues at the cutting edge of research and encourage vigorous debate of key current issues.
Important Dates

Registration open:

Call for Abstracts:

Call for Posters:
Now Open (accommodation can also be reserved)
January 2010
January 2010
The 2010 Committee is working hard on developing a program that looks at the cutting edge of science and
clinical management of MPS and related diseases. The Family Program will closely follow the Scientific Program
in areas of:

Diagnostics/Prognostics

Emerging therapies—cutting edge developments

Skeletal/soft tissue Pathology

CNS/Spinal Issues

Management Issues for MPS

Family and affected patients stories
Children’s Program
While parents are enjoying the Symposium the children will be having the time of their lives on an exciting and
stimulating children’s program that is being developed for the 2010 Symposium at some of Adelaide’s top attractions. A fully serviced crèche or day care centre will be available for children under the age of 4 or for those who
are too frail to participate in the planned activities.
Entry into Australia
All travelers, except New Zealand citizens, must obtain a visa or travel authority before traveling to Australia.
www.mps2010.com.au
WOULD YOU LIKE TO ATTEND THE 2010 MPS SYMPOSIUM?
A limited number of $500 travel bursaries will be available through the Canadian MPS Society.
Please contact us or visit our website for guidelines and registration forms. Application deadline: Feb 28, 2010.
Winter 2009
Page 15
Research & Clinical Trials
MPS I:
that has been observed in children with Hurler from the time
the patients are initially evaluated to the time they are one
Aldurazyme (laronidase) enzyme replacement therapy was
licensed for use by Health Canada on May 31, 2004, for long- year from transplantation. The study will involve four doses of
term treatment in patients with a confirmed diagnosis of MPS laronidase given during a lumbar puncture approximately
I, to treat the non-neurological manifestations of the disease. three months before transplantation, at the time of admission
to the hospital for the transplant, three months after the transFor more information on Aldurazyme, please visit
plant, and six months after the date of the transplant.
www.aldurazyme.com
Principal Investigator Dr. Paul Orchard can be contacted for
more information at 612-626-2961 or orcha001@umn.edu.
MPS I Intrathecal ERT for Spinal Cord Compression:
A one-year extension study has been approved. Enzyme replacement therapy (ERT) has been developed for MPS I. ERT MPS II:
helps many physical ailments due to the disease, but does not On June 15, 2007 Elaprase enzyme replacement therapy was
approved by Health Canada for the treatment of MPS II.
treat the central nervous system due to inability to cross the
blood brain barrier. The purpose of this study is to test deliv- Shire MPS II Intrathecal Study:
ery of ERT to the spinal fluid via intrathecal injection in paDr. Joseph Muenzer and will be conducting a Phase I/II
tients with MPS I. In this pilot study, recombinant human α- (Safety/Dosage) trial using intrathecal ERT for the treatment
L-iduronidase will be administered intrathecally once per
of MPS II, with a goal of preventing central nervous system
month for four months to individuals age 8 and older with
involvement. This study will take place at the University of
the Hurler-Scheie and Scheie forms of MPS I and spinal cord North Carolina in Chapel Hill, NC. If you are interested in
compression. If successful, intrathecal delivery could represent obtaining more information about the clinical trials, please
a practical, straightforward method of treating central nervous contact Dr. Muenzer (919)966-1447, or study coordinator
system disease due to lysosomal storage.
Heather Preiss at (919)843-5731 or hpreiss@med.unc.edu.
Primary Outcomes:
Safety of intrathecal enzyme treatment by blood and spinal
MPS III:
fluid tests each month; improvement in neurologic signs reDr. Grzegorz Wegrzyn of Poland has been researching sublated to spinal cord compression, by neurologic examination
strate reduction therapy and the effect of genistein on syntheand Japanese Orthopedic Association Scale each month; imsis of glycosaminoglycans.
provement in neurologic symptoms related to spinal cord
Shire Pharmaceuticals Group, as part of its research
compression, by subjective assessments and independence of
to evaluate new approaches to the problem of treatment of the
functioning scale each month; improvement in mobility, by
six-minute walk test each month; improvement in spinal cord central nervous system, is hoping to move its MPS III-A program forward. www.shire.com.
compression by MRI imaging and somatosensory evoked potentials at baseline and four months; improvement in lysosomal storage by spinal fluid glycosaminoglycan levels at
MPS IV:
each treatment.
In late 2008 the board of directors of Vivendy Therapeutics
Secondary Outcomes:
announced the cessation of Vivendy’s natural history program
Improvement in spinal fluid pressure, by opening pressure
as well as further medical activities related to the development
measurements at each intrathecal treatment; improvement in of enzyme replacement therapy treatment for MPS IV A.
hydrocephalus and other brain lesions by MRI at baseline and
BioMarin Pharmaceutical Inc. announced April 21,
four months.
2009 the initiation of a phase I/II clinical trial for BMN-110
Expected Total Enrollment: 10
or N-acetylgalactosamine 6-sulfatase (GALNS), intended for
Additional information can be obtained at
the treatment of MPS IV A.
http://www.clinicaltrials.gov/ct/show/NCT00215527?order=
Phase I/II clinical trials (safety) began in Q1 2009
1 or by contacting Principal Investigator Dr. Patricia Dickson with about 40 patients in the UK and there are about 20 enat 310-222-4145 or pdickson@ucla.edu.
rolled in the Phase II study. The Phase III trial will likely inMPS I Intrathecal ERT for Children Being Considered for
Transplant:
The University of Minnesota in Minneapolis has recently obtained FDA approval for the delivery of laronidase into the
spinal fluid of children with MPS I (Hurler syndrome) being
considered for marrow/cord blood transplantation. The goal
of these studies is to decrease the neurophsychologic decline
Page 16
clude 50-100 patients in several centres, and should begin
sometime in late 2010.
The link to BioMarin’s MPS IV A program website is
www.morquiobmrn.com - this site is a great place to look for
current information. It will be updated at least four times per
year, and of course, more often if there is significant news. In
addition, visitors have the option to register on the website to
The Connection
Research & Clinical Trials
receive news about updates via email.
Further information about MorCAP is also available
on ClinicalTrials.gov at this link;
http://www.clinicaltrials.gov/ct2/show/NCT00787995?term
=morquio&rank=2.
Patients or physicians who are interested in participating in MorCAP (see announcement on page 18) can contact the Montreal site’s study coordinator, Gail Ouellette at
gail.ouellette@mail.mcgill.ca or phone: 819-543-0550.
for more information.
If you have particular questions about operational
aspects of BioMarin’s trials, you can contact Candice Henkel
at chenkel@bmrn.com or 415-506-6973.
For additional medical questions related to MPS
IVA and the clinical details of BioMarin’s program, please
contact the Medical Director for BioMarin’s MPS IVA program, Celeste Decker, at cdecker@bmrn.com or 415-5066469.
MPS VI:
The U.S. Food and Drug Administration (FDA) granted marketing approval for Naglazyme(TM) (galsulfase), the first specific therapy approved for the treatment of MPS VI VI , on
June 1, 2005 .
Editor’s note: There is no word on when (or if) BioMarin will apply
for licensing approval for Naglazyme in Canada. For more information, please contact BioMarin Patient and Physician Support (BPPS)
at 1-866-906-6100 or bpps@bmrn.com.
MPS VII:
A gene therapy clinical trial for MPS VII, also known as Sly
syndrome, has been put on hold pending additional data.
Pompe Disease
Myozyme® (alglucosidase alfa), a recombinant form of human acid alpha-glucosidase (GAA), was approved by Health
Canada on August 14, 2006 for the treatment of patients
with Pompe disease (GAA deficiency). For more information
on Pompe disease, please visit Canadian Association of
Pompe’s (CAP’s) website: www.pompecanada.com.
Research Grant RFA
*****
The Canadian MPS
Society is requesting
applications for its 2010
$100,000 (over 2 years)
Research Grant for
research into MPS
Diseases
***
Grant guidelines and application forms
are available for download at
www.mpssociety.ca.
Application deadline: February 1, 2010
Funding date: March 30, 2010
DID YOU MISS THE VANCOUVER SYMPOSIUM? OR WERE YOU UNABLE TO CATCH
ALL THE SESSIONS WHILE YOU WERE THERE?
All scientific sessions were videotaped and are available for viewing, along
with their accompanying slideshows, at www.goldinfo.org.
To view the sessions on the GOLD website, you will need to register if you have
not done so before. Registering only needs to be done once. Click on “Video
Presentations” under “Education and Information” on the GOLD homepage’s left
sidebar. Then, click on “register here” in the first paragraph, which will open a
registration form. You will be asked to give your email address as a username,
choose a password, and select the organisation of which you are a member:
Please select Canadian Society for Mucopolysaccharide & Related Diseases Inc.
from the drop-down menu. GOLD will not give your information to third parties.
All presentations from the family sessions are available for download at www.mpssociety.ca.
Winter 2009
Page 17
Research/Clinical Trials
MORCAP CLINICIAL STUDY FOR
ÉTUDE CLINIQUE DU
MORQUIO SYNDROME TYPE A
SYNDROME DE MORQUIO TYPE A
The Montreal Children’s Hospital is the Canadian site for a
multinational clinical study of Morquio disease type A or
Mucopolysaccharidosis IV type A (MPS IVA) sponsored by
BioMarin Pharmaceutical Inc. The Morquio A Clinical Assessment Program (MorCAP) is designed to provide a fuller
understanding of MPS IVA syndrome in preparation of phase
III clinical trials of a potential treatment.
Patients of all ages are eligible for the MorCAP study. Participation will require a visit of 2 or 3 days to the Montreal
Children’s Hospital in Montreal. Travel and lodging expenses will be reimbursed. No experimental drug is administered during the visit. The study comprises a physical examination of the affected patients, blood and urine tests, a questionnaire concerning medical history, cardiac and respiratory
function tests, and endurance tests.
BioMarin is also conducting a Phase I/II study of an experimental enzyme replacement therapy for Morquio A. The
insights gained from theMorCAP study will help BioMarin
design the phase III trials. More information can be found at
http://www.morquiobmrn.com/ or at www.clinicaltrials.gov
If you are interested in participating in the MorCAP study at
the Montreal Children’s Hospital or if you want more information, contact the study coordinator, Gail Ouellette (email:
gail.ouellette@mail.mcgill.ca or phone: 819-543-0550.)
L’Hôpital de Montréal pour enfants est le site canadien pour
une étude clinique internationale de la maladie de Morquio
type A ou mucopolysaccharidose IV type A (MPS IVA). Cette
étude est commanditée par BioMarin Pharmaceutical Inc.
L’étude MorCAP (Morquio A Clinical Assessment Program)
a comme objectif de mieux comprendre le syndrome MPS
IVA en vue d’études cliniques phase III pour un traitement
potentiel.
Les patients de tout âge sont éligibles à l’étude MorCAP.
Leur participation nécessitera une visite de 2 à 3 jours à l’
Hôpital de Montréal pour enfants à Montréal. Tous frais de
transport et d’hébergement seront remboursés. Aucun
médicament expérimental ne sera administré durant cette
visite. L’étude comprend un examen physique, des tests de
sang et d’urine, un questionnaire sur les antécédents
médicaux, des tests de fonction respiratoire et cardiaque et
des tests d’endurance.
BioMarin évalue présentement un médicament expérimental
(thérapie de remplacement enzymatique) dans une étude de
Phase I/II. Les résultats de l’étude MorCAP permettront à
BioMarin de préparer les études cliniques de Phase III pour
ce médicament. Vous pouvez trouver plus d’information sur
ces études à http://www.morquiobmrn.com/ ou
www.clinicaltrials.gov.
Si vous êtes intéressé à participer à l’étude MorCAP à
l’Hôpital de Montréal pour enfants ou si voulez obtenir plus
d’information, contactez Gail Ouellette, coordonnatrice de
l’étude (courriel : gail.ouellette@mail.mcgill.ca ou téléphone :
819-543-0550).
Recruitment Open for New Study Led by Dr. Hal Siden
"Charting the Territory: Determining and Documenting Trajectories for Families Where a Child Has a LifeThreatening Condition" is a new multi-centre, longitudinal, descriptive study led by Dr. Rose Steele and Dr. Hal
Siden. Dr. Siden is the Medical Director of Canuck Place Children’s Hospice and a Clinical Associate Professor
at UBC.
This 5-year CIHR-funded study will identify and track children diagnosed with progressive neurological, metabolic, or chromosomal conditions. We will also follow their families to determine and document clinical progression of the child’s condition and the associated experiences of the family. Researchers aim to improve understanding of how to best care for these vulnerable children and their families.
Researchers have begun enrolling families through the Neurology, Biochemical Disease and Genetics clinic at
BC Children’s Hospital. For more information, please visit the study website:
http://www.chartingterritory.com/
Page 18
The Connection
Research/Clinical Trials
questions or sensitive stimuli will be used in the neuropsychological testing. MRI scans do not involve any radiation.
There are no known risks associated with magnetic resonance
scanning itself. We take care not to scan people with some
We are inviting individuals over the age of with MPS I, II or
types of metal in their bodies (from surgery or accident) and
VI to participate in a longitudinal research study seeking to
people with pacemakers or programmable shunts should not
better understand the brain basis for the learning difficulties
enter a magnetic resonance scanning facility. We will ask you
sometimes found in MPS disorders. You or your child must
detailed questions about these risks to ensure that it is safe
be over the age of 6 and able to cooperate in an MRI imaging
for the participant to enter the magnet. The blood draw has
study without any sedation. We are studying the central nervusual risks of bruising but it is only about the amount of two
ous system so that we can better understand the brain
teaspoons.
changes in MPS disorders to find better ways of treating these
problems.
Air and ground transportation and hotel for one night will be
provided to the University of Minnesota, Minneapolis camThe participant will be seen for two sessions in one day, one
pus, for two persons.
for neuropsychological testing that will last about two hours
to three hours, and the other for brain imaging which will last
You may contact or Kate Delaney at 612-625-1143
for one hour. Neuropsychological tests will include a brief
(delan011@umn.edu) or Elsa Shapiro at 612-625-1618
test of cognitive ability and attention, and several tests of
(shapi004@umn.edu) for more information. Thank you for
memory. Some of this testing will be done on a computer.
reading this description
This testing will be done in the Center for Neurobehavioral
Development at the University of Minnesota. You will also
Elsa Shapiro, Ph.D.
come to the Center for Magnetic Resonance Research. The
participant will be placed on a table in the scanner. Imaging Principal Investigator
Professor of Pediatric and Neurology
will require lying still for about 30 to 35 minutes. During
this time the participant can watch a video. We will be doing University of Minnesota
two procedures. One procedure is an MRI that allows us to
calculate the volume of various brain structures (volumetric
******************************
MRI). The other MRI procedure allows us to visualize the
structure in the connections between one part of the brain
Editor’s Note: The Hospital for Sick Children in Toronto will be a
and another (DTI – Diffusion Tensor Imaging). This study
site for this study with Dr. Julian Raiman acting as the Principal
will be different than clinical studies because we are using a
Investigator. Canadian patients are welcome to participate in this
more powerful magnet called a 3 Tesla scanner. Studies with study at the Toronto site or, if more convenient, at the Minneapolis,
a more powerful magnet are more sensitive to the details that Minnesota or Portland, Oregon sites.
we are investigating in this study and are just as safe as less
powerful magnets. We will also draw a small amount of blood
Please contact the Society for more information.
to measure new biomarkers that are being developed by Dr.
Clarke at the University of British Columbia.
Dear adult with or parent of a child with MPS I,
II, or VI:
None of these tests are in any way harmful. No embarrassing
EXPERIENCES OF FAMILIES OF CHILDREN WITH RARE DISORDERS
Principal Investigator: Prof. William H. McKellin, PhD, Anthropology, The University of British Columbia.
Dr. McKellin is inviting families to participate in this study of families of children with rare medical diseases or disorders.
This study aims to hear directly from you about health care, medical research, social services, and community support.
If you live in the Vancouver area and are interested in participating, please contact
Dr. McKellin at 604-822-2756 or mcke@interchange.ubc.ca.
Winter 2009
Page 19
2nd JAPAC Educational Forum for
The Taiwan MPS Society graciously hosted the 2nd JAPAC Educational
Forum for Families and Caregivers on MPS on October 9th and 10th
in Taipei. I was so pleased to be able to attend this very interesting meeting, where representatives from Asian countries—including Taiwan, Japan, China, South Korea, Malaysia, the Phillipines, Hong Kong, and
Singapore—discussed how to better support patients and families affected
by MPS diseases in their own countries, and how to better support each
other as they work as a collective, now called “The Asia-Pacifica MPS
Organization.” I was invited to give a talk about the Canadian MPS Society, and Barbara Wedehase (USA), Christine Lavery (UK), and Wendy
Boon (Australia) added to the international perspectives. The Forum
was held concurrently with the
12th Annual LSD Symposium
(a scientific meeting), and was
followed by the International
Conference of National Policies and Actions on Rare Diseases on October 12th, so altogether over four hundred delegates were in attendance and
took part in joint dinners and
outings, featuring a keynote
address by Dr. Roscoe Brady
and performances by extremely
talented Taiwanese dancers,
drummers, and singers. A
highlight was listening to the
incredible voices of the Taiwan Foundation for Rare Disorders’ children’s choir—their songs filled our hearts with hope.
Virginia Tsai, president of the Taiwan MPS Society did such a
Page 20
fabulous job of hosting us and I certainly appreciated having the opportunity to learn so much
about Taiwanese culture and to be inspired by all
the fabulous initiatives taking place across Asia in
support of those with MPS.
Kirsten
The Connection
Families and Caregivers on MPS
Wonderful memories from
Taiwan! Clockwise from top
left: TFRD’s Children’s Choir;
Kirsten with new friends from
China and Hong Kong, and with
TFRD’s founder Serena Wu;
holding the beautiful lanterns
that were presented as keepsakes of the conference. Bottom left: Virginia & Dr. ShuanPei Lin with Wendy, Barb &
Kirsten. Bottom right: Virginia
with her children. Opposite
page, left: William Luo giving an
inspiring speech about his life
with MPS II; right: the gong!
Winter 2009
Page 21
In the News
Particles Detected in Vials from Genzyme's
Plant Not Expected to Impact Aldurazyme or
Naglazyme
NOVATO, California, Nov. 13, 2009
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced
today that Genzyme notified the company that, in rare circumstances, it has detected foreign particles in some products
filled at the Allston Landing facility. To ensure that patients
are not exposed to foreign particles during product administration, Genzyme has issued a "Dear Doctor" letter to all physicians treating patients with enzyme replacement products
that it markets, including Aldurazyme. The letter states that
foreign particles have been found in some vials filled at Genzyme's Allston Landing facility and reminds physicians, as a
preventive measure, to use a 0.2-0.22 micron filter during
administration.
For Aldurazyme, this is essentially a reminder as the
Aldurazyme approved product label has always recommended
the use of a 0.22 micron filter when administering the drug.
Accordingly, BioMarin does not expect this letter to have any
substantial effect on the usage of Aldurazyme, or on the company's financials. Due to a mutual decision by BioMarin and
Genzyme, BioMarin has been transitioning Aldurazyme production to other fill finish facilities. The last fill finish of
Aldurazyme at Allston Landing was in September 2008.
BioMarin is committed to taking all efforts to maximize patient safety for all of its products. Similar to the Aldurazyme
label, the Naglazyme product label also recommends the use
of a 0.22 micron filter prior to administering the drug to patients. Additionally, BioMarin utilizes numerous process controls to minimize the presence of particles in its products,
including redundant filtration during the manufacture of
drug product.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The
company's product portfolio comprises three approved products and multiple clinical and pre-clinical product candidates.
Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed
and commercialized by BioMarin; Aldurazyme® (laronidase)
for mucopolysaccharidosis I (MPS I), a product which
BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of
Darmstadt, Germany. Other product candidates include PEGPAL (PEGylated recombinant phenylalanine ammonia lyase),
which is in Phase 2 clinical development for the treatment of
PKU and GALNS (N-acetylgalactosamine 6-sulfatase), which
is currently in Phase 1/2 clinical development for the treatment of MPS IVA. For additional information, please visit
www.BMRN.com. Information on BioMarin's website is not
incorporated by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the
manufacture and supply of Aldurazyme and Naglazyme and
the safety profile of these products. These forward-looking
statements are predictions and involve risks and uncertainties
such that actual results may differ materially from these statements. These risks and uncertainties include, among others:
the actual experience in manufacturing these products, future
unexpected adverse event reports, , and those factors detailed
in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained
under the caption "Risk Factors" in BioMarin's 2008 Annual
Report on Form 10-K. Stockholders are urged not to place
undue reliance on forward-looking statements, which speak
only as of the date hereof. BioMarin is under no obligation,
and expressly disclaims any obligation to update or alter any
forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of
BioMarin/Genzyme LLC.
SOURCE BioMarin Pharmaceutical Inc.
investors, Eugenia Shen, +1-415-506-6570, or
media, Susan Berg, +1-415-506-6594,
both of BioMarin Pharmaceutical Inc.
Just the tip of the iceberg - Hope for inherited childhood disorders
Top South Australian medical researcher honoured nationally
AUSTRALIAN INSTITUTE OF POLICY AND SCIENCE—November 9, 2009
Professor John Hopwood, South Australian Scientist of the Year 2008, will this evening follow in the footsteps of some of
Australia’s greatest medical researchers, including immediate past winner Professor Ian Frazer and Nobel Laureates Professors
Barry Marshall and Robin Warren, by being awarded the 2009 CSL Florey Medal.
Professor Hopwood today joins a cohort of extraordinary Australian medical researchers who are at the forefront of
breakthroughs globally, and who have dedicated their lives to basic research all the way through to making a difference to our
lives through prevention and treatment.
(Please visit our website to read the full press release.)
Page 22
The Connection
In the News
Swedish Driver Kalle Dejemyr
(22 years old with MPS type II)
Finishes 9th in the Swedish
Super Kart Championship
At the age of eleven Kalle Dejemyr was diagnosed with Hunter syndrome. He participated in the natural history and pharmaceutical trial in Mainz in 2005 and now receives
home treatment with Elaprase.
In the autumn of 2007 Kalle was
highlighted in the media when the hospital
management decided to discontinue his
treatment because of the cost. Kalle, his family and the MPS
society fought for his right to treatment and the decision was
changed after TV and the social minister was involved.
Medication has worked flawlessly and he now feels
almost completely healthy. Racing is one of Kalle's greatest
interests, where he can compete against others on the same
terms. Kalle is now driving/racing his second season in Super
Kart and has already achieved several podium finishes and
was ranked in 9th place in this year's Swedish Super Kart
Championship.
“I've had a tough season, have only been in goal in five of eight finals. It all started when my engine broke down on the very first contest. Have been forced to drive with spare engine, most of the SM
series. Finished in a good way with a 3rd and a 4th placing at the
last race. We have already started planning for next season.
I've been invited to run a race in Dubai 14-16 January 2010. It
would be a dream to drive in that race, but unfortunately we do not
have the financial means to participate. In Sweden is it difficult to
find sponsors, the hope is that I somehow can gather what is lacking
in the budget says Kalle.”
Hunter Disease eClinic
*****
This amazing new educational software, developed by Sick Kids’ Lysosomal Research Group and launched at
our International MPS Symposium, is now available through the Hospital for Sick Children’s website!
The direct links to the educational software are:
Ebook: http://www.sickkids.ca/research/lysosomalresearchgroup/contents/Hunter/Hunter_eBook.asp
Eclinic: http://www.sickkids.ca/research/lysosomalresearchgroup/contents/Hunter/Hunter.asp
Users need “Flash Player” installed on their computers in order to be able to view the program, and should be
reminded not to use the browser back button.
The Hunter eClinic can also be accessed from the Lysosomal Research Group’s web-site under
"NEW: Educational Resources" on the group home page: http://www.sickkids.ca/lysosomalresearchgroup/
Check it out!!!!
Winter 2009
Page 23
Fundraising—Toronto Waterfront Marathon
Team MPS
Continues Strong
In the early hours of September 27, 2009, a team
of dedicated individuals
assembled again to represent Team MPS at the
Scotiabank Toronto Waterfront Marathon.
We were blessed
with excellent (dry!)
weather, which was a
novelty this summer.
Leading the team with
an inspiring performance was Jenn Elder, our
sole half marathon runner. Also helping to represent was Kelly Leradza
from Genzyme, and all
five members of the
Byrne clan. A special
mention goes out to Team Billie: Robin Tewkesbury, Vanessa
Rooney, Tracy Davison and Shannon Boyd, all walking in memory of
Billie Tewkesbury. It was great to see Robin again!
I am
pleased to report
that our runners
managed to raise
$4,353.00 for the
Canadian MPS Society.
Way to go team!!!
Judy Byrne
Top: Team MPS gets ready to race! Left: Colin Byrne takes a one
kilometer running break on Dad Terry’s shoulders. Above: Jenn
Elder with daughter Anisa Mia (MPS I H/S), proudly wears her 1/2
marathon medal.
Page 24
The Connection
Fundraising—Toronto Waterfront Marathon
Clockwise from top
left: Team MPS believes! More MPS
Team members get
ready to race; Anisa
Mia Elder (MPS I H/S)
with Sarah Byrne (MPS
I H) enjoy a snack;
Team MPS members
getting close to the finish...wheelchairs in the
lead! Big smiles after
the race for all Team
MPS walkers and runners; Sarah Byrne
(MPS I H) runs with Dad
Terry. Thanks, team!
Winter 2009
Page 25
Fundraising—A Night of Enchantment
Right:
Emma-Rose
Grenon-LeMaitre
(MPS I H) cuddles
with her Dad Jim
LeMaitre
Above: Kirsten accepts $5000.00 cheque from Lara
Pietrolungo, organizer of A Night of Enchantment
On October 23, 2009, more than 300 guests attended the Emma-Rose Fund’s first inaugural fundraising gala, A Night of Enchantment, at Le Parc in
Concord, Ontario. A major accomplishment, this
first time affair raised over $50,000 to support families of children with Mucopolysaccharide (MPS)
diseases. To view the evening’s photos and the
Emma-Rose tribute video please visit
http://www.emma-rose.ca/index-3.html.
The Emma-Rose Fund’s main purpose is to increase
awareness of MPS diseases and raise dollars to support parents of children with MPS. As you might
know, proceeds from the gala will soon be donated
to your Family Assistance Program and to the Patient Amenities Program at the Hospital for Sick
Children. We feel that these two programs allow
parents across Canada to have direct access to funding to help care for their child with MPS disease.
This year, on behalf of the Emma-Rose Fund, it is
our pleasure to donate $5000 to the Canadian MPS
Society’s Family Assistance Program. All necessary
documents for issuance of tax receipts will be forwarded to you shortly. We thank you for you support and continued well wishes. We hope that this
is only the beginning of a great partnership.
Should you require any further information, please
don’t hesitate to contact us.
Warmest Regards,
Lara & Rudy Pietrolungo
Founders
Emma-Rose Fund
Left: Emma-Rose surrounded by family
and friends at the Emma-Rose Fund’s
inaugural fundraising gala
Page 26
The Connection
Fundraising—Walk with Nature
On September 26, 2009 we held our 6th MPS Walk with
Nature. The weather was great, the apple pie super
and the company fantastic.
Thanks so much to our supporters who have followed us from year to year and from town to town. We raised
$1010.00 in total for the Genetic department at the Montreal
Children's Hospital and The Canadian MPS Society. Our
faithful Dr. Serge Melancon and his family were present, and
as usual, the hospital Mascot. It always amazes me how rare
these diseases are, but what is more amazing is that out of
A beautiful day for the 6th MPS
Walk with Nature
total of $88.00
Our 18 speed bike was won by Lise Cournoyer from
Iron Hill Quebec. The bike was donated by Michele LeFevre
who works for The Montreal Children's Foundation; her
Monika Nelis-Dupont (MPS I H/S) enjoying the
walk with her energetic friends
about 35 people who attended our walk there were three MPS
families with us in the small town of Sutton Quebec. The
strength that MPS families bring to each other is a great blessing.
I would like to thank all the donors who donated
items for our Chinese auction. Our 50/50 draw was won by
Henry St-Amour from Sutton and he donated it right back: a
Monika was all tuckered out and sat in her pink
limo for the 2 km. ride back to the hall.
friend had won it at a previous event.
Thanks to Sylvie for organizing the Chinese auction
once again. It was a lot of fun and we had over 30 auction
items, and Lisa Phaneuf did a great job of keeping track of the
item winners. I was very happy to see Jim and Adele LeMaitre, now from Quebec, come out and join our walk. And last
but certainly not least, we truly do appreciate the support of
the Royal Canadian Legion who donated the hall, their help,
their time.
Nathaniel Phaneuf (MPS II), with Dad Russell &
sister Chelsea chat with Caramel along the way.
Winter 2009
THANKS TO ALL!
The Nelis-Dupont family
Page 27
Fundraising——Hope Shines Bright Gala
Celebrating The Sanfilippo Children’s
Research Foundation’s
10th Anniversary at the
Hope Shines Bright Gala April 2009
The room was sparkling with shimmering glitter, dazzling
lights, twinkling disco balls, shining mirrors, and almost
700 radiant smiles! The Hope Shines Bright 10th Annual Evening for Elisa reflected everyone’s excitement as the Foundation marked ten years of successful fundraising, awareness
building and promising research that’s producing measurable results.
This Gala was also a celebration of Elisa’s fifteenth birthday. By the mid teens, many Sanfilippo children have been confined to their beds or, worse, have
Elisa Linton (MPS IIIB) with her family on stage at the
passed away from the illness. We were all so happy to see
Hope Shines Bright Gala
Elisa return again this year as belle of the ball and even
appear at the podium along with the rest of the Linton
family.
Just over $190,000 was raised in this one evening
alone—a sum that makes the Foundation proud and grateful, especially considering so many people are feeling the
pinch of tough economic times. Returning as Emcee for
this special year was CFRB morning man Bill Carroll. Herbie Kuhn, the on-court voice of the Toronto Raptors, was
our lively Auctioneer for the tenth year running. And guest
speaker Simon Ibell, who has a related metabolic condition
(MPS ll - Hunter’s Syndrome), shared his unique story of
believing and never giving up hope in finding a cure.
To mark the 10th year, 12 individuals were recognized for their outstanding donation and contribution of
professional services and unique gifts to the Foundation for
each of the past ten years. Without their help since incepThe room before the arrival of the almost 700 guests
tion of the SCRF, we would not be able to contribute 97
cents of every dollar to research. Our sincerest appreciation was extended to:
Dr. Joe Clarke, Paul Mosey, Wendy Rae, Barry
Pettit , Sarah Double, Kim Aikenhead , Tracy Galvin, Herbie Kuhn, David Kraulis, Mark Boehmer, Tracy Keenan,
David Levy & Bill Carroll.
Heartfelt thanks also goes to our generous and
loyal donors for contributing hundreds of amazing auction
items, over 80 volunteers who helped Elisabeth and Randall pull the evening together, and to our guests for keeping precious hope alight. Thanks, too, to everyone who sent
in donations in lieu of attending—these funds make a huge
difference in our mission to find a treatment or a cure for
Sanfilippo.
Stay tuned for more details of next year’s gala event which will
celebrate Elisa’s “Sweet 16.” It’s never too early to get involved. If
you would like to join the Gala Organizing committee, please
Simon Ibell (MPS II) speaks to gala guests
contact Elisabeth at alifeforelisa.org or 416-223-1911.
Page 28
The Connection
Fundraising
Canadian MPS Jeans Day at Cove Cliff Elementary School in North Vancouver
Thanks so much to everyone who donned their jeans and
supported Canadian MPS Jeans Day at Cove Cliff Elementary
School on October 2nd. Nicklas was diagnosed with MPS I
in 2001 when he was a kindergarten student at Cove Cliff,
and we have been so touched over the years by the outpouring
of support from our school community. Even though Nicklas
is now a high school student at Seycove Secondary School,
Cove Cliff continues to hold a Jeans Day annually, and this
year raised over $1,200.00!
Thanks so much, Cove Cliff, for caring so much
about Nicklas and all kids with MPS!
Kirsten Harkins
Above left: Jansen Harkins (far left, top row) and
his grade seven class; above: Jonas Harkins (2nd
from left, top row, beside his teacher Mrs. McAllen)
and his grade 3/4 class on Jeans Day.
FUNDRAISING COOKBOOK
With the support of local merchants, Mary Nelis, mother of Monika Nelis-Dupont (MPS I H/S), will
be working on a cookbook this winter. The cookbook will raise funds for the Canadian MPS Society.
Please contribute by sending your favourite recipes to her at mary.nelis@sympatico.ca. The cookbooks will be available for purchase in 2010.
Friends of Ferrari Luncheon
The Harkins family was pleased to represent the
Canadian MPS Society at an annual charity luncheon
hosted by Vancouver-based “Friends of Ferrari.”
The Society has been honoured to receive
funds through Friends of Ferrari’s mini-Ferrari races,
and we were pleasantly surprised when Franco
Corona, founder of Friends of Ferrari, announced
that we will be receiving additional funds at year-end.
Many thanks to Franco and his wife Suzann,
and all the other dedicated volunteers who have
helped them raise $1,257,000 for 42 charities.
For more information, visit www.friendsofferrari.com.
Franco Corona poses with Jonas, Kirsten & Nicklas Harkins, and a beautiful red Ferrari!
Winter 2009
Page 29
Fundraising– Canadian MPS Jeans Day
Help bring awareness of
MPS Diseases into your
community!
Hold a Canadian MPS Jeans Day
on any day of the year— just
follow these simple steps:
 Submit a proposal to your school's principal or
your employer
 **email Kirsten (kirsten@mpssociety.ca) or Jill
(jill@mpssociety.ca) for a word template**
 Email or call the head office to let us know how many people you anticipate will be participating.
You will receive a Canadian MPS Jeans Day package with enough stickers for your event. Packages
will also contain 2 posters, 5 colour brochures (you will have to photocopy enough brochures to distribute at your event.), and a donor information sheet to record the names and addresses of those
wishing to receive income-tax receipts for donations of $10.00 and over.
 Fill in the event date and expected donation amount on your posters (the expected donation is up
to your discretion depending on the location: we recommend between $1.00—$5.00) and advertise
Canadian MPS Jeans Day at your school or workplace. Email us for an 8x11 poster PDF if you
would like to print out additional posters.
 Organize volunteers to collect donations and distribute stickers and brochures on the morning of
your event.
 Ensure those who donate $10.00 or more fill out the donor form completely if they wish to receive
income tax receipts.
 Take a photo at your event to share with our members in the next issue of the Connection!
 Submit your donations, donor forms, photos, and a brief write-up, with photos if possible, about
your event within 30 days of the event date.
Canadian MPS Jeans Days are easy to hold and are excellent opportunities to raise awareness and funds
for the society. Many of our members have found them to be extraordinarily helpful in educating
school communities about our children's unique conditions. Please consider holding a Canadian MPS
Jeans Day at your child's school or at your workplace.
Would YOU like to organize a fundraising event for the Canadian MPS Society?
Look for great fundraising ideas in these pages, or contact us at info@mpsociety.ca!
Page 30
The Connection
On-going Fundraising
On-going Fundraisers:
More Great MPS Fundraising Ideas:
Used Stamps:

Sign up through your United Way Donor Choice
Program. Designate The Canadian MPS Society as your
“OTHER” choice by providing our name, address and
Charity # 129030409 RR0001. Double your support by
enrolling the Society in matching gift programs, available through many companies.

Organize a walk or run

Have a garage sale

Organize a Sports Tournament

Participate in Craft Sales

Organize a Canadian MPS Jeans Day

Host a party: i.e. Pampered Chef, Discovery Toys,
Partylite.

Organize a dinner or barbeque

Remember, when you sponsor a fundraiser you are not
only raising money to help find a cure but you’re also
educating the public about MPS.
Please save your stamps and mail to our stamp collector:
Laura Wassilyn
5 Kitson Drive
Scarborough, ON M1M 3C7
TIPS ON MAILING STAMPS:
Please trim stamps leaving 1/4
inch of paper around them. Any with tape or damaged perforations should NOT be submitted as they cannot be sold.
Thanks for your support!
Fundraising Reminders:
Contact the office and request an event proposal form.
Fill out and submit the form so that the head office knows
the details regarding the events you are holding.
Don’t forget to submit a brief article, with photos if possible,
for the Connection about your fundraising success stories and
suggestions—they are terrific resources for other families
planning events.
Thanks for your enthusiastic support!
The Canadian MPS Society’s tribute card program is a special
way of paying tribute to a friend or a loved one.
Send an MPS tribute card for any occasion as a meaningful gift to support children affected
with MPS: to welcome a new baby, to celebrate a birthday or anniversary, to memorialize the
passing of a friend or relative or to say thank you to someone special. MPS tribute cards may
be ordered by making a contribution of $10.00 or more. Contact the office with your request:
sympathy or tribute cards are sent out the same day and income tax receipts are issued.
Do you need respite care?
Caring for an exceptional child can be exhausting—The Maxwell
Alexander Settari Fund has generously donated funds to our
Family Assistance Program specifically for respite care.
Interested members should submit a Family Assistance Program
application form, found on page 34, and contact Kirsten for guidelines.
Winter 2009
Page 31
Society Merchandise
CANADIAN MPS SOCIETY GOLF SHIRTS!
AVAILABLE IN MEN’S XXL, XL, L, M, & S, AND WOMEN’S
(fitted style with buttons) L, M, & S.
$40.00 (includes shipping)
ALSO AVAILABLE ARE A LIMITED NUMBER OF CHILDREN’S T-SHIRTS
(These shirts come in Youth L, M and S and are for sale for $12.50 (including shipping).
TO ORDER, visit our online store at www.mpssociety.ca
or call us at 604-924-5130 or on our toll free number 1-800-667-1846
NEW! “Expression of Hope II”
art cards are now available!
These inspiring cards come in a pack
of 20 original designs (including the
beautiful piece to the left).
View all selected pieces as well as all
submissions to Expression of Hope II at
www.expressionofhope.com.
Artist: Happy
Country: Taiwan
Condition: Fabry
Order your art cards for $25.00 per package
(including shipping) by visiting our online
store at www.mpssociety.ca, or calling
604-924-5130 or 1-800-667-1846.
“I dream of having a pair of my own wings, to fly
freely beyond the sky, so no matter how far I go,
I won't feel tired. I close my eyes and happily
spread my wings heading to my Paradise.”
ORD ER YOUR
CANADIAN MPS SOCIETY
AWARENESS BRACELETS!
Our bracelets are royal blue and inscribed with “BELIEVE - MPSSOCIETY.CA”
Our awareness bracelets come in three sizes: large, medium, and small and are available
for a minimum donation of $2.00 per bracelet, plus shipping.
Please place your order through our online store at www.mpssociety.ca
or call us at 604-924-5130 or 1-800-667-1846 and help spread the word about MPS diseases!
Page 32
The Connection
The Canadian Society for Mucopolysaccharide & Related Diseases Inc.
2010 Membership Form
To register or renew your membership, please submit this form or go towww.mpssociety.ca (please log in to your account when renewing)
First/Last Names: _________________________________________________________________________________
Address: _______________________________________________________________________________________
City: __________________________________ Province: _________________ Postal Code: __________________
Telephone: ________________________________ E-mail: ______________________________________________
Affected Family:
Relative/Professional/Other:
Out of Country:
______$25.00
______$30.00
______$35.00
AFFECTED CHILD’S NAME(S) AND
Enclosed membership payment:
Enclosed donation:
Total:
DATE OF BIRTH
MEMORIAL DATE
$ ________
$ ________
$ ________
SEX
SIBLINGS’ NAMES
DIAGNOSIS/
MPS DISORDER
Would you like your name to appear in our Parent Referral Directory? Yes ______
No ______ Initial consent:__________
Would you like to have your affected child’s name appear on the Birthday/Memorial page? Yes ______ No ______
If so, please fill in all information in the above chart, as you would like it to appear.
I am willing to allow the Society to publish pictures of my living/deceased child(ren) for posters, advertising, booklets, brochures,
and the MPS website. Yes ______ No ______ (Please send/email clear photos. We will return photos if requested.)
Signed:______________________________________________ Date:_____________________
Please send information on MPS Diseases to the following professionals:
Name:_______________________________________________________________
Address:_____________________________________________________________
IF YOU HAVE AN AFFECTED CHILD AND CANNOT AFFORD THE $25.00 MEMBERSHIP FEE,
PLEASE INFORM THE OFFICE AND WE WILL SEND YOU COMPLIMENTARY NEWSLETTERS.
The Canadian MPS Society is committed to providing support to fami- Please make cheques payable to:
lies affected with MPS and related diseases, educating medical profesTHE CANADIAN MPS SOCIETY
sionals and the general public about MPS, and funding research so
that one day cures will be found for all types of MPS and related disPO Box 30034, RPO Parkgate
eases. Members will receive:







Four newsletters per year (Spring, Summer, Fall, Winter)
Our Family Referral Directory
Our Annual Report
Access to our Family Assistance Program
Advocacy support
All new publications printed by the Society
Invitations to attend family meetings and conferences
Winter 2009
North Vancouver, BC V7H 2Y8
Please charge my credit card in the amount of $______________
Credit Card #________________________________________
Name on card ________________________________________
Expiry Date __________Signature________________________
Registered Charity # 12903 0409 RR0001
Page 33
FAMILY ASSISTANCE PROGRAM
FUNDING APPLICATION
Date:_________________
Name:________________________________________________________________________
Address:_____________________________________________________________________
_____________________________________________________________________________
Phone: ___________________________________
Email: ___________________________________
Name(s) of affected child(ren):___________________________________________________
Amount of funding requested: ______________
Please describe what the funds will be used for: ____________________________________
_____________________________________________________________________________
_____________________________________________________________________________
_____________________________________________________________________________
Will the funds being requested be used to pay for part of a larger project or piece of equipment?
(eg. home renovations, wheelchair-accessible van, etc.)
Yes________No_________
If you answered yes to the previous question:
What is the estimated total cost of the project/equipment? _____________
Please list other funding agencies you have applied to for funding for this project/equipment,
and funds received, if any:
_______________________________________________________________________
_______________________________________________________________________
□ Estimate(s) included. (Please see guidelines for information on required estimates.)
Signature______________________________________________Date___________________
Thank you for submitting your Family Assistance Program funding application. The Executive Director or a member of the Board of
Directors will contact you as soon as possible to inform you of the Board’s decision regarding your application.
Please visit www.mpssociety.ca or email kirsten@mpssociety.ca for complete Family Assistance Program guidelines.
Page 34
The Connection
The Canadian MPS Society’s Publications
Canadian MPS Society booklets and merchandise can now also be ordered through our online store at www.mpssociety.ca
NUMBER
OF COPIES
NAME OF BOOKLET
PRICE
PER COPY
Family Resource Book - English or French*
4.00
Daily Living with MPS and Related Diseases
2.00
MPS I (Hurler/Scheie Diseases) - English or French *
2.00
MPS II (Hunter Syndrome)
2.00
MPS III (Sanfilippo Syndrome)
2.00
MPS IV (Morquio Syndrome)
2.00
MPS VI (Maroteaux-Lamy Syndrome)
1.00
Mucolipidosis II (I-Cell Disease) and III
1.00
Fucosidosis
1.00
Mannosidosis
1.00
Aspartylglucosaminuria
1.00
Mucolipidosis IV
1.00
Multiple Sulfphatase Deficiency
1.00
Tay-sachs & Sandhoff Disease
1.00
Hearing impairment in MPS Children
1.00
Is your child Having an Anaesthetic?
1.00
Bone Marrow Transplants in MPS Children
1.00
The Pattern of Inheritance
4.00
Choices – When your child is Dying - English or French*
7.00
Video—A Roll of the Dice
10.00
TOTAL
PRICE
*All booklets are in English with the exception of the Family Resource Book, MPS I Booklet & Choices. Please circle language.
PLEASE ADD $2.50 FOR SHIPPING. (For 10 or more booklets add $5.00.)
Name: __________________________________________________________________________________________________
Address: ________________________________________________________________________________________________
City:______________________________________________Province ________________Postal Code: ____________________
Telephone: ( _____) _________________
# of Booklets:
_________________
Shipping & Handling: _________________
Send your order and cheque to:
The Canadian MPS Society
PO Box 30034, RPO Parkgate
North Vancouver, BC V7H 2Y8
For payment by credit card, please call our head
office at (604) 924-5130 or 1-800-667-1846.
Winter 2009
Page 35
S
MPS Society Logo Wear

Indigo Sweatshirts
$30.00

Jean Shirts
$30.00

Indigo T-shirts
$20.00

Children’s t-shirts
$15.00
M
L
XL
Plus $5.00 for shipping & handling (per item)
Total Enclosed__________
Page 35
Season’s Greetings
to all members of the Canadian MPS Society!
On behalf of the board of directors and all of us at the Society,
we wish you health and happiness in 2010
This year’s winner of our Holiday Art Contest is by Monika Nelis-Dupont, MPS I H/S, age 10.
Her artwork has been featured on the Canadian MPS Society’s Holiday Greeting Cards for 2009.
PO Box 30034, RPO Parkgate
North Vancouver, BC V7H 2Y8
Tel: (604) 924-5130 / 1-800-667-1846
Fax: (604) 924-5131
www.mpssociety.ca