Featured Blog Posts – October 2014 Archive (5)


Staff
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…

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Added by PPMD on October 27, 2014 at 4:23pm — 2 Comments


Staff
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 wal…

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Added by PPMD on October 23, 2014 at 9:00am — 1 Comment


Staff
News from World Muscle Society: Day 4

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…

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Added by PPMD on October 13, 2014 at 9:30am — No Comments


Staff
News from World Muscle Society: Days 2 & 3

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…

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Added by PPMD on October 10, 2014 at 9:40am — No Comments


Staff
News from World Muscle Society Meeting (10/8/14)

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…

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Added by PPMD on October 9, 2014 at 9:00am — No Comments

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