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Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans.
Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and to provide the seed funding necessary to accelerate his work in this company, we could not be more thrilled. This is a true testament to the power of venture philanthropy and I want to thank each and every donor and supporter who has believed in our work, and we are grateful to Dr. Olson for his dedication to Duchenne patients. Although we still have a long way to go before we confirm this therapy will be safe and effective in humans, this dramatic dystrophin restoration offers hope to all families affected by this disease.
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Pharmacokinetic Properties of Chronic Administration of Eteplirsen in the Treatment of Boys with Duchenne Muscular D...Objective: The objective of this analys...
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Critical scientific aspects that make gene therapy viablehttps://player.vimeo.com/video/275297686...
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Nonprofit CureDuchenne is Encouraged to See Gene Editing for Duchenne Advance Through Vertex’s Acquisition of Exon...CureDuchenne, a nonprofit global leader ...
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Kathryn Wagner, MD, PhD – Progress in Better Treatments for Muscular Dystrophyhttps://www.youtube.com/watch?v=0yC4_lEn...
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Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug ApplicationOn February 14, 2019, Sarepta Therapeuti...
