Duchenne Research Update: October 2016
So much has happened in recent weeks in the Duchenne space that it can be hard to keep up. PPMD’s Abby Bronson, SVP, Research Strategy provides a roundup of the latest Duchenne research news.
End Duchenne eNews: A Battle Won, But the War Continues
Catch up on the latest research and community updates in this month's End Duchenne eNews.
PPMD Demands Anthem Reconsider Coverage of Approved Drug
Health insurer Anthem recently released a medical policy statement on Exondys 51 stating that the product is "investigational and not medically necessary." PPMD has taken action to demand Anthem reconsider coverage.
Connect with PPMD in 2017
PPMD has been busy putting together an exciting list of events for 2017 that will continue to connect and inform our Duchenne community. We hope you can make one of these great events in the coming year!
PPMD Awards $239,000 Research Grant to Dr. Terence Partridge of Children’s National Medical Center
This grant to Dr. Terence Partridge of Children's National Medical Center will help Dr. Partridge and his team continue their work in optimizing exon skipping technology for future clinical trials in Duchenne.
Calling All Adults with Duchenne/Becker – Join the 2017 PPMD Adult Advisory Committee (PAAC)!
If you are an adult member of our Duchenne/Becker community, please consider lending your time, talent, and vision to paving our pathway forward! Apply to join the PAAC by Friday, October 28th.
[Webinar Takeaways] Preparing for Access to Approved Therapies
PPMD recently began a webinar series on access an exciting new chapter for our Duchenne community.
[Upcoming Community Call] Sarepta and Summit
Sarepta Therapeutics and Summit recently announced a license and collaboration agreement, which grants Sarepta the right to commercialize Summit's utrophin modulators in Europe should they receive approval. PPMD will be hosting a community update call with Sarepta and Summit on Monday, October 10 at 12:00pm EDT to answer any of your questions.
Anthem and Exondys 51 (Eteplirsen)
PPMD is deeply troubled to learn that health insurer Anthem will not be covering Exondys 51 (eteplirsen). PPMD has reached out to Sarepta and will assist the company however we can to appeal this decision. Our community is still in the very early days of this drug approval and we remain optimistic that Anthem will revise their decision following further review and discussion. PPMD is tackling this issue head on so that every single person amenable to exon skipping 51 has access to this therapy.
[Webinar Recording] SareptAssist Patient Services Overview
On September 26th, Parent Project Muscular Dystrophy and Sarepta Therapeutics hosted an informational webinar about SareptAssist, Sarepta's patient support program.
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.
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.