Changing the Paradigm: The Eteplirsen Ad Comm
There is no way around it, today was hard. Like you, we are dumbfounded by the closing votes and discussion. But a decision has not been made – these were recommendations. And we believe that the number of questions with mixed votes represent opportunities for us to change minds and continue to influence the agency.
April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information
The Center for Drug Evaluation and Research (CDER) plans to provide a free of charge, live webcast of the meeting.
PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews
Great news as Congress continues to communicate urgency to the FDA to apply flexibility in review of Duchenne therapies, thanks to our PPMD community!
PPMD Names Children’s Hospital Los Angeles 10th Certified Duchenne Care Center
PPMD is thrilled to announce our 10th Certified Duchenne Care Center -- Children's Hospital Los Angeles!
PPMD's Every Single [One] Tour Launched in Baltimore!
On Saturday, Duchenne community members from six states gathered together at Kennedy Krieger in Baltimore to hear the latest developments on clinical trials, clinical care practices, carrier programs, legislative and regulatory efforts, IEPs and school advocacy, physical therapy, genetic testing and more.
Senate Sends Strong Letter to FDA about Duchenne Therapies
With this letter now sent, both the House & Senate have communicated a strong message to the FDA about the need to use every possible tool at their disposal to speed access to promising Duchenne therapies.
End Duchenne eNews: Coming to a Town Near You
Catch up on the latest research and community updates in this month's End Duchenne eNews.
PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters
We are pleased to release the Community Report for this study, which takes a deeper look into the results. Later this year, an extended version of the report data will be published in multiple publications.
PPMD's 2016 Connect Conference Agenda Now Available
PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June 26-29. Make sure you register you and your family before the Early Bird Registration Rate ends April 15th!
ACTION ALERT: Please urge Your Senators to Sign Letter to FDA
Please urge your Senators to sign this important letter. Patients need and deserve therapeutic options and the FDA must use the tools they have been given to expedite these options.
How You Can Help with the Upcoming Ad Comm for Eteplirsen
We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. The momentum is tremendous but we need YOU to take action leading up to the big day.
Webinar Recording: BMS Anti-Myostatin Adnectin Program
PPMD and Bristol-Myers Squibb (BMS) recently hosted a webinar to discuss the BMS Anti-Myostatin Adnectin Program.
PPMD Community Survey about the Akashi HT-100 study
If you or your child were in the Akashi HT-100 clinical trial that was recently suspended, please take our survey. Responses are needed by April 10th.
Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy
There has been a flurry of press recently on gene therapy in Duchenne and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne.
PPMD is Initial Partner in OnPAR
PPMD is proud to participate in OnPar which provides access to peer-reviewed research proposals that directly relate to Duchenne research. OnPAR will provide us with an efficient way to solicit early stage and translational Duchenne research proposals, see the breadth of research in Duchenne, and gain access to novel research strategies.
DuchenneConnect & UCLA Recruiting Patients for Genetic Modifiers Study
The Genetic Modifiers Study is trying to figure out what genetic changes are playing a role in the difference of symptoms that we see in Duchenne. The more people we have participate, the more powerful the study.
How You Can Participate in the Eteplirsen Ad Comm
The Duchenne community is working closely together to prepare for the April 25th Advisory Committee Meeting for Sarepta’s therapy eteplirsen. Learn about the different ways you can become involved in this historic event.