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…
Added by PPMD on October 27, 2014 at 4:23pm — 2 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…
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…
ContinueAdded 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…
ContinueAdded 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…
ContinueAdded by PPMD on October 9, 2014 at 9:00am — No Comments
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