Our Call to Anvisa: All Brazilian Children Over 7 Should Receive Elevidys Gene Therapy
Duchenne Muscular Dystrophy (DMD) is a rare, life-limiting genetic disorder that primarily affects boys, leading to progressive muscle weakness and degeneration. The disorder has...
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 Accessing 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...