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October 2014 Blog Posts (9)


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
Enterovirus

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…

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Added by Kathi Kinnett on October 25, 2014 at 11:30am — No Comments


Staff
Notes from FDA’s Presentation at NORD Summit

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…

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Added by Ryan Fischer on October 23, 2014 at 9:30am — No 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


Staff
Calling All Adults with Duchenne/Becker – This Means YOU!

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…

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Added by Annie Kennedy on October 6, 2014 at 10:14am — No Comments


Staff
Early Pulmonary Care Webinar Summary & Recording - 9/24/2014

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…

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Added by Kathi Kinnett on October 3, 2014 at 9:00am — No Comments

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