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...
In Which Countries Is Elevidys Approved? Is Elevidys Approved In Europe?
Elevidys is the only approved gene therapy in the World for Duchenne muscular dystrophy and received accelerated approval in the US in June 2023,...
First Girl in the World to Receive Elevidys Gene Therapy: Raniya Scott
When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), a group of diseases that...