The Jett Foundation Fighting Duchenne Muscular Dystrophy

Save Your Seat For DMD History – April 25th

March 10, 2016

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The eteplirsen AdComm has been rescheduled for Monday, April 25, 2016.  The meeting will be held at the College Park Marriott Hotel and Conference Center in Hyattsville, MD.  If you wish to attend the AdComm, please register here.

 The FDA Advisory Committee Open Public Hearing (OPH) is our community’s opportunity to share expertise, evidence, and patient experience data from those in the eteplirsen trials, and from Duchenne experts, that eteplirsen is safe and effective and warrants accelerated approval.  PPMD, the Duchenne Alliance, the Center for DMD at UCLA, Jett Foundation, and the Race to Yes have all been working together with input from experts on the FDA approval process to determine what types of messages will be most impactful from our community. 

Due to the high interest in this historic event, many families and patients have expressed interest in speaking during the OPH. We invite the Duchenne community to join our Coalition for a collaborative webinar early next week to explain the strategies behind the OPH speaking assignments, congressional engagement, and media outreach. Please stay tuned for more information on this upcoming educational webinar.

As you know, the highest priority speakers are those patients, families, clinicians, and researchers who can share their direct experiences with eteplirsen and provide context to the scientific data being presented during the sponsor presentation. Our coalition is working together to make sure those people are prioritized and registered.

If more people register than can be accommodated in the two hours allotted for the OPH, the FDA will use a lottery system to determine which people get to speak. Without coordination, we would be leaving the speaking assignments to chance, and world-renowned Duchenne experts and patients on drug could be left without an opportunity to speak. We don’t want that to happen.

However, everyone in the Duchenne community has an important role to play in this AdComm and in the approval of eteplirsen. If you have a message you want to share, please contact us at MakeDuchenneHistory@gmail.com and we will help make sure that you make an impact, whether it is at the OPH in a group of OPH speakers who have a similar story or message, on the Hill, in the press, or elsewhere.

Previously awarded travel scholarships to Ad Comm will be honored. Please register here to attend the Ad Comm and you will be contacted shortly.

 More information on other events planned around the Ad Comm will be shared soon.  Thank you for your continued support! 

TAKE A STAND. FILL A SEAT.  

RAISE YOUR VOICE.

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