Posts By: theracetoyes

Can’t Be in DC? Watch the FDA AdComm Streamed Live.

On Monday, April 25th, the Duchenne Community will come together in an overwhelming show of strength at the FDA Advisory Committee meeting with one message:  eteplirsen is safe, effective, and deserves approval. We thank the nearly 900 members of our community who made the trip. For those of you who cannot attend in person, there are… Read more »

Ad Comm Travel FAQ’s

Who is eligible for travel assistance? ANYONE with a friend, family member, or loved one with Duchenne who is coming to the April 25th Advisory Committee meeting is eligible for travel assistance.  Assistance is not limited to those with a formal role in the AdComm. What is covered? The Jett Foundation and UCLA will cover… Read more »

Details & Guidance on Submitting Written Testimony to the FDA Advisory Committee

Why Are Written Comments Important? How Are They Utilized? Written comments are compiled and shared with the FDA Advisory Committee members prior to the date of a product’s/ company’s Advisory Committee meeting. Thus, written comments are a critical part of helping to provide context to the Advisory Committee. Who Should Consider Submitting Written Comments? Any… Read more »

The FDA Ad Comm: What types of information should be included in written testimony?

What types of information should be included in written testimony? Your direct experiences with Duchenne, and if applicable, with eteplirsen specifically. Testimony from those who have been involved in the clinical trials as a participant, parent of a participant, clinician, researcher, or community member and have first-hand knowledge of how eteplirsen has impacted the daily… Read more »

Important Update: Eteplirsen NDA

After a meeting yesterday with the FDA, Sarepta Therapeutics plans to submit a rolling New Drug Application (NDA) for eteplirsen beginning next week and wrapping up the application by the middle of this year. This is great news, and it would not have happened without all your work and support, so THANK YOU. We have… Read more »