News Update - eNews

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.
June 19, 2013 | Learn more

Jun 11 Webinar

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.
June 13, 2013 | Watch the Webinar

News Update - Action Alert

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!
May 29, 2013 | Learn more

News Update - eNews

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.
May 16, 2013 | Learn more

News Update - Webinar

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.
April 30, 2013 | Learn more

News Update - Clinical Trial

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!
April 30, 2013 | Learn more

News Update - Duchenne Central

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.
April 29, 2013 | Learn more

Putting Patients First

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.
April 22, 2013 | Learn more

 
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Pat's BlogExpanding the Scope of Acceptable Endpoints in Accelerated Approval

Why the FDA should expand the scope of acceptable endpoints used to approve drugs for Duchenne.

May 30, 2013 | Read More

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