Blog

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 »

The Race to Yes: Where Do We Stand Today?

July 2012 Drug company Sarepta announces that after 36 weeks of dosing, DMD participants using eteplirsen outperformed the placebo. The company had demonstrated three months earlier that eteplirsen results in the production of significant levels of dystrophin, the protein boys with Duchenne are missing. December 2012 Sarepta requests a meeting with the FDA to determine… Read more »

The FDA’s Roller Coaster Ride for DMD Families

For months, the Duchenne community expected eteplirsen and drisapersen to be reviewed by an Advisory Committee on back-to-back days in November 2015. Now the FDA has delayed eteplirsen again until next year. This is unacceptable. The Race to Yes and patient advocacy organizations sent an urgent letter to the FDA demanding answers. As of November… 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 »

One Year Later: An Important Infographic Update

One year ago, you helped us rally more than 106,000 people to sign a White House petition urging approval for safe and effective treatments for Duchenne. Today, a full year later, we are still fighting for the fast and efficient regulatory approach that our children deserve. The FDA’s actions make it clear that they do… Read more »

Enough is Enough

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… Read more »