End Duchenne eNews: Appreciating Parents
Catch up on the latest research and community updates in this month's End Duchenne eNews. Also meet our Featured Voice for June, 20-year-old Ben Dupree.
Webinar: Benefit/Risk Survey Results
On Wednesday, June 12 PPMD hosted a webinar to discuss preliminary results from our ongoing benefit/risk survey. We have collected meaningful data about the Duchenne population, in the hopes of advancing the development of new treatments.
MD-CARE Act: Time to Reach Back Out
Hundreds of PPMD advocates have already met with, phoned, or emailed your Members of Congress to ask them to cosponsor the MD-CARE Act legislation. However, some still have not yet signed on. It’s time to reach back out and remind them how important the MD-CARE Act has been to our families!
2013 Connect Conference: Agenda Now Available
PPMD’s Annual Connect Conference will be here before you know it!
This summer we return to Baltimore, Maryland at the beautiful Marriott Waterfront with our most exciting and important agenda yet focusing on Duchenne: Better, Faster, Now!. Check out who is presenting when.
Drisapersen Update webinar with GSK
The purpose of the webinar is to update the Duchenne community about Phase II clinical trial results for GSK’s drisapersen drug for the treatment of Duchenne. GSK investigator John Kraus will present and answer submitted questions. PPMD, CureDuchenne, and MDA will co-sponsor this webinar.
DuchenneConnect Year-End Report
We had a busy and exciting year as we continue to expand our services to patients and families, as well as to researchers and clinicians. A big “thank you” to everyone who registered on DuchenneConnect in March - we had 92 new patient registrations, our highest month ever!
New Mobile App: Locate Duchenne Clinical Trials
PPMD is thrilled to launch Duchenne Central! This new app for your smartphone allows you to search for Duchenne clinical trials and clinics near you or near a specific location. You can also keep track of the latest news, bookmark items, and share with family and friends. Keep your family in the know in real time.
New PPMD White Paper
PPMD is proud to share an advance copy of Putting Patients First: Recommendations to speed responsible access to new therapies for Duchenne, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne.
We are optimistic that Putting Patients First will provide guidance for the FDA and other regulatory agencies as they continue to understand Duchenne and this community’s need for accelerated approval.
Why the FDA should expand the scope of acceptable endpoints used to approve drugs for Duchenne.
May 30, 2013 | Read More