Treating the Whole Disease
Transcription
Treating the Whole Disease
Duchenne muscular dystrophy T reating the W hole Disease DUCHENNE IMPACTS: 1 in every 3,500 live male births What Is It? is the most common and lethal muscle disease in boys. 24,000 300,000 boys in the U.S. boys worldwide Impact on the Body DUCHENNE IS A PROGRESSIVE, MUSCLE-WASTING DISE ASE. It results from a defective gene responsible for producing the key muscle protein, dystrophin. Without dystrophin, cells easily become damaged and die, resulting in heart and breathing failure. What is CureDuchenne? CureDuchenne is a nonprofit that raises awareness and funds research to find a cure for Duchenne. POSSIBLE LEARNING AND COGNITIVE DIFFICULTIES CUREDUCHENNE’S COMPREHENSIVE RESEARCH STRATEGY: treatments to reduce disease severity Slow progress of the disease Affected boys usually are diagnosed before age 5 ... DECREASED HEART FUNCTION CARDIOMYOPATHY LEADS TO HEART FAILURE drug repositioning New use of existing drugs protein replacement Restore dystrophin and complementary proteins CUREDUCHENNE’S IMPACT WEAKENS DIAPHRAGM REQUIRES VENTILATOR IN TEENS LEADS TO PNEUMONIA ... confined to wheelchairs by age 12 ... LOSS OF MUSCLE MASS WEAKNESS INFLAMMATION FIBROSIS ....and most don’t survive their mid-20s. Number of research projects advanced into human clinical trials with CureDuchenne’s support. 7 2014 Year expected to see first drugs approved. Two drug development programs supported by CureDuchenne will be the first to seek approval for treatment of Duchenne. $100 MILLION Amount of money leveraged from government agencies and pharmaceutical companies to fund research leading to a cure. 100 MILLION Number of people reached through awareness campaign. Based on current scientific knowledge, Duchenne could become treatable for this generation of boys. BRITTLE AND WEAK We just need the funding now. Help us CureDuchenne Now. www.cureduchenne.org
Similar documents
Duchenne Muscular Dystrophy Market Treatment, Symptoms, Pipeline Review, H1 2016
Global Markets Direct's, 'Duchenne Muscular Dystrophy - Pipeline Review, H1 2016', provides an overview of the Duchenne Muscular Dystrophy pipeline landscape. Visit Us @ http://www.radiantinsights.com/research/duchenne-muscular-dystrophy-pipeline-review-h1-2016
More informationThe Duchenne Diagnosis: What Does it Mean?
good clinical care • Caution patients to be wary of any conversation that starts “But you have to…” • Every family is different, and will have to find
More information