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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 18, 2016 Category: Neurology Source Type: news
PPMD Demands Anthem Reconsider Coverage of Approved Drug
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 14, 2016 Category: Neurology Source Type: news
Connect with PPMD in 2017
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 14, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 4, 2016 Category: Neurology Source Type: news
[Webinar Recording] SareptAssist Patient Services Overview
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - October 3, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - September 15, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - September 14, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - September 9, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - September 1, 2016 Category: Neurology Source Type: news
PPMD Visits South Africa to Help Spread Global Knowledge of Duchenne Care
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 31, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 29, 2016 Category: Neurology Source Type: news
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 24, 2016 Category: Neurology Source Type: news
PDUFA VI & Its Impact on Duchenne
PPMD has submitted comments we hope will ensure that PDUFA VI best reflects the needs of our community. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 19, 2016 Category: Neurology Source Type: news
End Duchenne eNews: Every Single Step
Catch up on the latest research and community updates in this month's End Duchenne eNews. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 16, 2016 Category: Neurology Source Type: news
[Webinar Recording] PTC Therapeutics Provides Regulatory Update on Translarna - August 2016
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. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 16, 2016 Category: Neurology Source Type: news
