Dear FDA officials, Please do not delay your decision about Sarepta’s new drug application any longer. Eteplirsen is producing dystrophin in 100% of the boys…
On Monday, Charley turned 13 years old. Birthdays cause emotional turmoil for all Duchenne parents I know. We’re deeply grateful for every single day with…
Last week, the New York Times reported disappointing news. A promising treatment for Duchenne failed to meet its primary endpoint in a Phase 3 clinical…