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...
Ask the DMD Associations in Your Country: When Will Duchenne Treatments Come to Our Country?
We continue to voice the problems of DMD patients from many countries around the world. Duchenne muscle patients report that DMD associations in their...
Will Elevidys Receive Approval from the European Medicines Agency (EMA)?
Elevidys, a gene therapy developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD), is currently under review by regulatory bodies in...