Help us bring the ABLE Act to the Floor!
The Achieving a Better Life Experience (ABLE) Act (S. 313/H.R. 647) has been 8 years in the making and has the support of more than 85% of Congress; please help us get it over the finish line! We need you to make 4 easy phone calls, tweets, and posts to ensure ABLE gets scheduled for a vote!
PPMD Names Baltimore’s Kennedy Krieger Institute a Certified Duchenne Care Center
Kennedy Krieger Institute in Baltimore, Maryland is the fifth center to be certified by PPMD, recognizing the Institute’s dedication to improving care for people living with Duchenne.
End Duchenne eNews: Patients Are Waiting
Catch up on the latest research and community updates in this month's End Duchenne eNews!
Webinar Recording & Summary: Duchenne Pulmonary Care for Tweens and Teens
A summary and recording of last week's pulmonary care webinar is now available. Thank you to Dr. Jonathan Finder (Children’s Hospital of Pittsburgh) and Dr. Oren Kupfer (Children’s Hospital Colorado) for such an informative hour.
Patients Are Waiting...
Thanks to the dozens of you who participated in PPMD’s benefit/risk project and took the time to share your story, we have been able to compile your responses and will be providing the FDA with “Patients Are Waiting…” These are your stories, your messages to the Agency demanding that they use all the tools at their disposal to make decisions efficiently. We don’t have time.
PPMD Submits Letter to FDA Regarding Latest Sarepta Update
Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons.
PPMD-Funded Gene Therapy Study Improves Walking Ability in Muscular Dystrophy
Dr. Jerry Mendell of Nationwide Children’s Research Institute recently presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for follistatin can improve performance on the 6 minute walk test in study participants with Becker muscular dystrophy. This is the first time a gene therapy has demonstrated an improvement in function in a muscular dystrophy.
Webinar Recording: Efficacy of Catena®/Raxone® (Idebenone) on Respiratory Outcome in Duchenne
The recording of our webinar with Santhera Pharmaceuticals is now available. Prof. Gunnar Buyse (University Hospitals Leuven, Belgium; PI of the study) and Thomas Meier PhD (CEO of Santhera Pharmaceuticals) presented data of the Phase III DELOS trial of Catena®/Raxone® (Idebenone) in Duchenne.
Calling All Adults with Duchenne/Becker!
PPMD is seeking Adult members of our Duchenne/Becker community to serve on the newly-forming PPMD Adult Advisory Council (PAAC). Please consider lending your time, talent, and vision to paving our pathway forward. Application deadline: Friday, October 17th.
Webinar Recording: Prosensa Update about Drisapersen & Other Programs
On October 2nd UPPMD hosted a webinar with Prosensa. Prosensa answered questions from the Duchenne community and gave an update on drisapersen and other Prosensa Duchenne programs.
Webinar Recording & Summary: Early Pulmonary Care
A summary and recording of our early pulmonary care webinar is now available. Thank you to Dr. Hemant Sawnani, Dr. Richard Shell, and Dr. Oscar Henry (“Hank”) Mayer for such an informative hour.
President Signs MD-CARE Act Amendments Into Law
Thank you again to everyone in this community who helped advocate for the passage of this critical legislation! We couldn't be more proud to be on your team!