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Next Thursday, an FDA advisory committee will meet to consider whether to approve a treatment for Duchenne muscular dystrophy. The last time an FDA advisory committee met to review a therapy for this fatal childhood disease, things did not go smoothly. — Portrayed in the media as a heated showdown between the FDA and desperate… Read more »

Dear Friends,   As we set out on the New Year, we’re looking back on what our community has accomplished and focusing forward on how to make more progress. 2016 was a monumental year, with the first-ever drug approval for Duchenne. We are proud to have been a part of that and grateful to everyone… Read more »

Today, the first safe and effective treatment for Duchenne muscular dystrophy—Eteplirsen—was approved. This is a proud day for every member of our Duchenne Community who worked for this moment and this historic victory—the experts who weighed in with the FDA, the community who rallied, and most of all the 12 boys in this trial who… Read more »

Sarepta Therapeutics announced this morning that the FDA has indicated it will not reach a final decision on eteplirsen by the May 26th deadline.  We understand and share the frustration many of you feel that the FDA still hasn’t made a final decision on eteplirsen despite strong scientific evidence that supports approval. We have already… Read more »

By next Thursday, the FDA will make a decision on approval for eteplirsen. Whether the FDA’s answer on eteplirsen is “YES,”or “YES, BUT…,” or “NO,” the race is far from over. If eteplirsen is approved, we will celebrate this historic milestone of the first ever therapy for DMD. For the 87% who cannot benefit from… Read more »

It’s not emotion, it’s science. We thank the scientific community from around the globe who came to Washington DC to defend the science behind eteplirsen. We encourage the @US_FDA to honor FDASIA, and fully utilize the tools Congress has given them to allow us to have this drug. ‪#‎MakeDuchenneHistory‬

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 »

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 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! 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 »