Upcoming Webinar: What is an Ad Comm?
PPMD will present a webinar, WHAT IS AN AD COMM?, on Wednesday, September 2 at 1pm eastern.
August 24, 2015 | Learn more
Upcoming Webinar: Getting Involved with
Coach to Cure MD!
PPMD will present a webinar, Getting Involved with Coach to Cure MD on Wednesday, August 26th at 1pm eastern.
August 19, 2015 | Learn more
End Duchenne eNews: Fighting Duchenne With Awareness
Catch up on the latest research and community updates in this month's End Duchenne eNews!
August 15, 2015 | Learn more
PPMD’s DuchenneConnect to Receive $1.4 Million Funding Award from PCORI
DuchenneConnect, led by PPMD, has been approved for a three year, $1.4 million funding award by PCORI, the Patient Centered Outcomes Research Institute! This funding award allows us to continue our work as a member of PCORnet, the National Patient-Centered Clinical Research Network.
August 12, 2015 | Learn more
What is an Ad Comm?
Ad Comm. It’s a term our community will be hearing a lot this fall as companies get closer to the possible approval of treatments for Duchenne. But what is an Ad Comm and how does it involve and affect you or your child? PPMD will host a webinar on September 2 at 1pm eastern to discuss this topic in detail. Guests will include FDA staff.
August 11, 2015 | Learn more
Imperatives for DUCHENNE MD: A simplified guide to care for Duchenne published in PLOS
PPMD is proud to present a published version of Imperatives for DUCHENNE MD a one page snapshot of Duchenne care created by a small group of international experts from PPMD, UPPMD, and TREAT-NMD. We hope you continue to find this a useful resource and we thank PLOS for helping us share this information with the medical community.
August 11, 2015 | Learn more
One month away from World Duchenne Awareness Day!
People around the world will help participate in the second annual World Duchenne Awareness Day on September 7th. There are a variety of ways you can get involved with this international effort how will you raise awareness in your community?
August 7, 2015 | Learn more
PPMD Submits Comments on FDA's Draft Guidance on Duchenne
We are coming to the end of the 60 day open comment period for the FDA’s draft guidance on Duchenne. PPMD submitted our comments to the agency yesterday. While we hope you agree with our response, we respect alternate viewpoints. If you disagree with any comments or have other issues you think the guidance needs to address, we urge you to submit your opinions as well through the FDA public comment mechanisms. This community-wide effort continues to be acknowledged as precedent setting and a variety of rare disease groups are following in our footsteps. We have so much to be proud of and look forward to next steps with the FDA.
August 6, 2015 | Learn more
Action ALERT: Urge your Senators to Cosponsor S. 1597, the Patient-Focused Impact Assessment Act
Call and email your Senators today and urge them to sign on as a cosponsor of S. 1597, the Patient-Focused Impact Assessment Act, before they go on recess on August 8th. This is critical legislation for our community.
August 5, 2015 | Learn more
Different Together: PPMD Announces the Duchenne Regulatory Science Consortium
PPMD is thrilled to announce the launch of the Duchenne Regulatory Sciences Consortium (D-RSC) with the Critical Path Institute! Each instance of Duchenne seems to be as unique as the person it affects. The D-RSC will begin by aggregating a wide array of clinical data for analysis by consortium members, with the goal of developing a disease progression model that can be used in the development of new treatments, and reduce the amount of time it takes to get them to people living with Duchenne.
August 3, 2015 | Learn more
Changes to Deflazacort Ordering Process (via Masters Pharmaceuticals)
Masters now has an increasing supply of Deflazacort (a steroid available to treat Duchenne outside the US). However, due to recent regulatory changes, changes have been made to the ordering process to assure that the company is able to continue to supply Deflazacort to US families.
July 27, 2015 | Learn more
PPMD's 2015 Connect Conference Resources & Recordings
Presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download.
July 21, 2015 | Learn more
PPMD Awards $70,000 Grant to Talem Technologies as Part of Robotics Initiative
PPMD is proud to award Talem Technologies with a grant for over $70,000 to support a pilot study of their X-Ar exoskeletal arm technology. This grant is a direct result of your donation this spring for our Robotics Initiative. We believe in the X-Ar arm and other technologies that we believe will maintain muscle function and foster independence. Thank you for supporting our robotics program and congratulations to Talem Technologies!
July 20, 2015 | Learn more