Duchenne Muscular Dystrophy (DMD)

A Landmark Development for the Duchenne MD Community

DMD: Prosensa Pursuing Path Forward for Drisapersen

Update (May 1, 2014): For encouraging results from an open-label, extension study of drisapersen (all participants receive the drug in an open-label study), see Prosensa's May 1, 2014, press release.

MDA Thrilled FDA Will Consider Accelerated Approval for New Muscle Disease Drug Aimed at DMD

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.

Shooting for Hope


Bryson is an honor roll student at school. His favorite subjects are reading, spelling and social studies, but sports are his true passion. He collects sports memorabilia. Bryson served two years as MDA’s National Goodwill Ambassador from 2011 – 2013. He and his parents traveled throughout the United States attending events, meeting with sponsors and spreading the message of MDA’s mission. He appeared on the Labor Day Telethon as a co-announcer for two years.

DMD: Phase 2 Drisapersen Results Encouraging

Update March 27, 2014: Prosensa and United Parent Projects Muscular Dystrophy jointly presented an educational webinar for patients and families affected by Duchenne muscular dystrophy on March 25, 2014.

DMD, BMD: Ataluren Did Not Receive Early Approval in Europe

PTC Therapeutics Provides Update On CHMP Opinion For Conditional Approval Of Ataluren For Nonsense Mutation Duchenne Muscular Dystrophy

DMD: What's Next for Drisapersen?

Eteplirsen Data Still Strong: Treated Boys with DMD Stable at 120 Weeks