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 »
Monthly Archives: April 2016
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 »
Share This 25-Second Video
We’re just three short weeks away from the critical FDA advisory committee meeting to evaluate eteplirsen on April 25th. This is a critical milestone for boys with Duchenne. Our community is coming together to ensure that the committee gets a full understanding of the science behind this safe and effective treatment. We need a roomful… 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 »