Organization Urges FDA to Use the Evidence Presented by Dozens of Scientists, Physicians, Patients and Advocates as Required by 2012 FDASIA Act for Conditional Approval Experts Remind Duchenne Community that Ad Comm Vote on accelerated approval was very close (6 yes-7 no); FDA Can Still Make Right Decision to Approve the Drug Physicians and scientists… Read more »
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 »
Race to Yes Calls on FDA Advisory Committee: Carefully Consider Data and Recommend Duchenne Drug Approval
Race to Yes Calls on FDA Advisory Committee: Carefully Consider Data and Recommend Duchenne Drug Approval Based on Data and Direct Experience of Children and Clinicians WASHINGTON, April 22, 2016 (GLOBE NEWSWIRE) — In what is predicted to be the largest turnout in history at an FDA Advisory Committee meeting, more than 800 clinicians, researchers,… Read more »
Please call your Senator and ask them to sign the Wicker-Klobuchar Duchenne letter to the FDA!
Please call your Senator and ask them to sign the Wicker-Klobuchar Duchenne letter to the FDA! Read the full text of the letter here. The following Senators have signed as of Friday, April 8: Wicker (MS), Klobuchar (MN), Warren (MA), Rubio (FL), Markey (MA), Schumer (NY), Bennet (CO), and Barrasso (WY). Click here for a… 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 »
FDA Advisory Committee Reviews First Duchenne Treatment for US Approval
Tomorrow is a big day. A special advisory committee convened by the FDA will spend all day reviewing the first Duchenne treatment up for approval in the US. By the end of the day, the “ad comm” will answer questions FDA reviewers have posed to help them decide whether to approve the treatment. Regardless of… 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 »
Duchenne Families to the FDA: Enough is Enough. No More Regulatory Delay
IT’S PAST TIME TO SAY YES TO SAFE AND EFFECTIVE TREATMENTS FOR DUCHENNE MUSCULAR DYSTROPHY The Problem Duchenne Muscular Dystrophy is an always fatal disease for which there is no currently available treatment. It affects about 1/3500 boys born every day. Boys are diagnosed between the age of 2 and 5; they will stop walking… Read more »