Everyone is well aware that we are in the thick of flu season. Recently, PPMD board member, and Duchenne parent, Ellen Wagner, got first-hand experience dealing with the scariness of the…

Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research (CDER) presented the opening keynote at NORD's Rare Diseases and Orphan Products Breakthrough Summit in Washington, DC on October 21.



Her remarks focused on how the patient advocacy community can most effectively…

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 wal…

Late Breaking Talks:

PPMD-funded gene therapy study improves walking ability in muscular dystrophy

Dr. Jerry Mendell of Nationwide Children’s Research Institute presented data from a ground-breaking PPMD-funded…

PTC Therapeutics Symposium – “Improving Outcomes for the Future: Making sense of the nonsense mutations in Duchenne muscular dystrophy”

PTC Therapeutics gave an overview of the data supporting the recent accelerated approval…

Sarepta held a symposium on the role of dystrophin production in disease-modifying treatments for Duchenne. Some highlights:

We heard from Dr. Francesco Muntoni from the Dubowitz Neuromuscular Centre about how much…

Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in policy and program efforts?

PPMD is seeking Adult members of our Duchenne/Becker community to serve on the…

The lungs require the strength of many muscles to help a person to breathe and cough. As people with Duchenne grow older, the respiratory muscles, like the skeletal muscles, become weaker. Like the heart, every person with Duchenne will have pulmonary issues of some…