We Call on Health Ministries and Pharmaceutical Manufacturers to Take Action on Duchenne Treatments
It’s heart-wrenching to think that children in today’s world, in countries across the globe, are losing their lives far too early simply because they...
Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments
While DMD families struggle to restore their children to health, pharmaceutical companies whose primary mission is to heal children are focused on year-end profits....
The Challenge of Getting DMD Gene Therapy: Is Geography Destiny?
Why are DMD gene therapies so expensive? Over the last decade, significant advancements have been made in the development of gene therapies for DMD,...
Call from Families to European Medicines Agency (EMA): Re-examine Ataluren (Translarna)
Three parents representing 800 families and their children affected by Duchenne Muscular Dystrophy and a nonsense mutation are calling on the European Medicines Agency...
Brazilian Children Over 7 and Their Families Demand Elevidys Gene Therapy from Anvisa
ELEVIDYS, the only currently approved gene therapy for DMD, is approved in Brazil for children ages 4 to 7. However, according to the decision...