Defeat Duchenne Family Forum with Jacques P. Tremblay
Key Note Speaker: Dr. Jacques P. Tremblay Presented by PTC Therapeutics and in partnership with Jesse’s Journey and La Force DMD, this unique educational event provides the opportunity for [...]
Viltolarsen under priority review by the FDA
The U.S. Food & Drug Administration (FDA) had accepted the filing of a New Drug Application (NDA) under the priority review for viltolarsen in patients with Duchenne Muscular Dystrophy (DMD) [...]
RARE DISEASE DAY – February 29, 2020
February 29, 2020, will be the thirteenth international Rare Disease Day coordinated by EURORDIS. On and around this day hundreds of patient organizations from countries and regions all over the [...]
Translarna™ Preserves the ability to walk for longer in children with DMD
PTC Therapeutics Announces First Publication of Real-World Data Showing Translarna™ (ataluren) Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy [...]
