Upcoming Webinar 9/29 - Navigating Access to Therapies Once Approved
Join us Thursday, September 29th at 1pm ET for the first in an ongoing informational webinar series to help empower you with tangible action items and resources to prepare for therapy access.
FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy!
PPMD is beyond thrilled at today’s historic news from the FDA announcing the accelerated approval of Sarepta Therapeutic’s eteplirsen, now the first approved drug for Duchenne in the U.S.! Eteplirsen is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with Duchenne.
End Duchenne eNews: Every Single Coach
Catch up on the latest research and community updates in this month's End Duchenne eNews.
PPMD Continues to Lead Work on Critical Duchenne Access Issues
With the promise of drug approvals on the horizon, access to these therapies will be critical to our Duchenne community. PPMD has been leading work related to access issues on multiple fronts.
Action Alert: 21st Century Cures Legislation
September is THE critical month to get 21st Century Cures/ Senate Innovation legislation over the finish line. Tell Congress why this matters so much to our community.
PPMD Awards $200,000 Grant to Joshua Selsby, PhD of Iowa State University
Dr. Selsby is exploring the potential benefit of combining lisinopril with quercetin to see if the two compounds in combination would be more effective than either alone.
September 7: World Duchenne Awareness Day
For the third year in a row, the global Duchenne community will come together on September 7 for World Duchenne Awareness Day.
PPMD Visits South Africa to Help Spread Global Knowledge of Duchenne Care
Thank you to the Muscular Dystrophy Foundation of South Africa for helping us take another step in the global effort to end Duchenne in support of the upcoming World Duchenne Awareness Day on September 7!
Essential Back-To-School Resources for Duchenne
PPMD has compiled a list of helpful resources you can utilize to help ensure a smooth start to the school year.
One Month Away from the 9th Annual Coach to Cure MD!
Join us September 24th as thousands of coaches from hundreds of universities wear the Coach To Cure MD patch on their sleeve to raise awareness and tackle Duchenne.
PDUFA VI & Its Impact on Duchenne
PPMD has submitted comments we hope will ensure that PDUFA VI best reflects the needs of our community.
PPMD Sends Letter to FDA Regarding Duchenne Therapies
PPMD has sent a letter to the FDA requesting updates on various regulatory actions of tremendous importance to the Duchenne community.
[Webinar Recording] PTC Therapeutics Provides Regulatory Update on Translarna
PPMD recently hosted a webinar with PTC Therapeutics to discuss the latest news regarding Translarna and the path forward for the therapy with the FDA.
Help the Powers Family #TackleDuchenne!
The 9th Annual Coach To Cure MD on September 24 is shaping up to be our biggest year yet!
Duchenne mom Kim Powers shares why this is the year you should get in the game and help us #TackleDuchenne.
2016 Connect Conference Resources & Recordings
We are excited to share with you presentations from PPMD’s 22nd Annual Connect Conference! We will continue to update this page over the coming weeks with slides and video content.