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...
Turkish DMD Patients Who are Eligible for Exon 51 Skipping Treatment (Eteplirsen) Request Their Medications from the Ministry of Health
The DMD Dayanisma Community, founded by families of children with Duchenne Muscular Dystrophy and followed by many DMD patients in Turkey, wants children who...