It is hard to imagine anything worse than receiving the diagnosis of Duchenne. The dates and odysseys that brought us into this community differ, but the fervor we share to slow and stop the progression of this disease as it marches through our children is unparalleled in any…Continue
We're excited to start off PPMD's Every Single [One] Tour: Kansas City, MO event weekend with a Professional Day for nurses and physical therapists! Thank you to Claudia Senesac (University of Florida) for joining PPMD's Kathi Kinnett at Children's Mercy Hospital to help us educate physical therapists, nurses, and students in the lifelong care needs of people living with Duchenne. We're looking forward to seeing our families tomorrow! Visit…See More
Sarepta Therapeutics and Summit recently announced a license and collaboration agreement, which grants Sarepta the right to commercialize Summit's utrophin modulators in Europe should they receive approval. This includes ezutromid, which is currently in the Phase 2 PhaseOut DMD trial. Learn more by listening to the recording of PPMD's community update call with Sarepta and Summit.See More
Your presence in Washington, DC this February is a critical part of the Duchenne community's strength and impact going forward. Please consider joining us February 12-14 as we urge Congress to continue to make Duchenne a priority!See More
2017 PPMD Advocacy Conference
Registration is now open on PPMD's website: http://www.parentprojectmd.org/Advocacy2017 ------ Please join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy. 2016 marked a tremendous victory in our collective history, the first FDA approval of a Duchenne therapy – Exondys 51. It…See More
Join us for our first DuchenneConnect Twitter chat on Thursday, October 20 from 1-2:00 PM EST. We will be using the hashtag #DuchenneConnectTC. Our special guests will be Dr. Richard Wang and Dr. Stan Nelson, both from UCLA. Dr. Nelson and Dr. Wang have been partners with the registry for many years and will answer questions about how they have used DuchenneConnect data and ways we could think about using it in the future. We will also have DuchenneConnect Family Advisors Alan Chaluet,…See More
Catabasis provides a quarterly update on their MoveDMD trial with edasalonexent (CAT-1004) in Duchenne, highlighting the completion of enrollment for Part B of the MoveDMD trial and other recent news and events. We appreciate these updates and are proud to have a been an early supporter of this promising therapy.
RESEARCH UPDATE: So much has happened in recent weeks in the Duchenne space that it can be hard to keep up. While approvals and regulatory actions have dominated the press, there is also a lot of pre-clinical and early stage clinical research happening behind the scenes with great potential. PPMD’s Abby Bronson, SVP, Research Strategy provides a roundup of the latest Duchenne research news.…See More
Coach To Cure MD is not just a day, it’s a season. And it’s not just football coaches, it’s communities! Thank you to the teachers and students of Acton Elementary in Granbury, TX who helped the Madrigal family #TackleDuchenne by wearing special Coach To Cure MD shirts in honor of 6-year-old Jax the day of their event!