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.
End Duchenne eNews: Going the Distance to End Duchenne
Catch up on the latest research and community updates in this month's End Duchenne eNews!
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 last week's 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!
The MD-CARE Act: Reflections & Projections
Sometimes it is worth taking a moment to reflect so that as we continue to plan ahead we can learn from not only our mistakes, but our victories. Our advocacy initiatives continue to grow and we won’t slow down. We can’t. And we hope you’ll join us as we continue to use our voice in D.C.
Duchenne Newborn Screening
Our Duchenne community has therapies in today’s pipeline that will be most effective when administered early. Our formalized efforts to move towards Duchenne newborn screening began several years ago; our community push begins now.
BREAKING NEWS: MD-CARE Act Passed!
Today – September 18, 2014, our Duchenne community once again made history by demonstrating that nothing can stop us. Today, the MD-CARE Act Amendments of 2014 passed! WE DID IT!!
PPMD Names Seattle Children's Hospital Certified Duchenne Care Center
Seattle Children's is the fourth center to be certified by PPMD, recognizing the hospital's dedication to improving care for people living with Duchenne.
PPMD Welcomes Annie Kennedy as Senior Vice President of Legislation & Public Policy
We are thrilled to welcome Annie Kennedy, our new Senior Vice President of Legislation and Public Policy! Annie joins PPMD after being with the Muscular Dystrophy Association (MDA) for 25 years, first as a camp volunteer and as an employee for the last 16 years. Annie’s focus will be on advancing public policy, addressing transition issues for people with Duchenne, and working on newborn screening issues.