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…
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 enterovirus and rhinovirus when her son Tim fell ill. I asked Ellen to…Continue
Added by Kathi Kinnett on October 25, 2014 at 11:30am — No Comments
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 accelerate therapy development along the drug development…
Added by Ryan Fischer on October 23, 2014 at 9:30am — No Comments
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…Continue
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 study demonstrating that a modified virus carrying the gene for…Continue
Added by PPMD on October 13, 2014 at 9:30am — No Comments
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 for Translarna® in Europe and answered questions from…Continue
Added by PPMD on October 10, 2014 at 9:40am — No Comments
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 dystrophin is expected to be needed to stabilize the muscles and result…Continue
Added by PPMD on October 9, 2014 at 9:00am — No Comments
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 newly-forming PPMD Adult Advisory Council…Continue
Added by Annie Kennedy on October 6, 2014 at 10:14am — No Comments
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 type.
The goal of pulmonary care is to allow both the prevention and management of…Continue
Added by Kathi Kinnett on October 3, 2014 at 9:00am — No Comments