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2018 Annual Conference Resource Round Up

PPMD’s 2018 Annual Conference Resource Fair Providers Each year we welcome specialized resource providers from around the country to interact with us at PPMD’s Annual Conference. We so appreciate the time and investment from all…

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15th Annual Jumping Jack Charity Golf Tournament Another Huge Success to Help End Duchenne

The 15th Annual Jumping Jack for Duchenne MD Golf Tournament, Wine Tasting, Dinner & Silent Auction that took place on June 18thin Westminster, CO was another huge success! The Knight Family has raised over $530,000 over…

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Summit Therapeutics Announces PhaseOut DMD Did Not Meet Primary Endpoint

We are disappointed to learn that Summit Therapeutics plc today announced that their PhaseOut DMD trial has not met its primary or secondary endpoints after 48 weeks of treatment of ezutromid in patients with Duchenne….

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Vamorolone (VBP15) Top Line Data Shows Improvements in Strength and Endurance in Boys with Duchenne

ReveraGen BioPharma, with the Cooperative International Neuromuscular Research Group (CINRG), conducted an international clinical trial of vamorolone at 11 CINRG sites in 6 countries (US, Canada, Australia, Israel, UK, and Sweden). ReveraGen reports that the…

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#PPMDConference: Live Stream the Latest Duchenne Research News and Updates

Research. There is a momentum in the Duchenne research pipeline, the likes of which we have never seen before. Join us at PPMD’s Annual Conference, Thursday, June 28 – Saturday, June 30 to find out…

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2018 #PPMDConference Live Stream: Care & Advocacy Updates You Won’t Want to Miss!

With PPMD’s Annual Conference quickly approaching, we wanted to make sure you are aware of the incredible care and advocacy content that will be covered while we are in Arizona this week! Care It’s at…

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PPMD’s Decode Duchenne Program Serves 1,000th Patient

PPMD is pleased to announce the 1,000th patient application to our Decode Duchenne program. Decode Duchenne is a nationwide program to assist individuals with Duchenne muscular dystrophy in accessing genetic testing, interpretation, and counseling. The…

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FDA’s Patient-Focused Drug Development Initiative: What Have We Learned?

PPMD’s SVP of Legislation and Public Policy, Annie Kennedy, joined a distinguished panel discussion convened by FasterCures about the impact of Patient Focused Drug Development on the Duchenne community and how it has become a…

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Positive Preliminary Results from the First Three Children Dosed in Phase 1/2a Gene Therapy Micro-dystrophin Trial

Sarepta Therapeutics announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne. Ryan…

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Join the 2018 #PPMDConference Live Stream!

Next week we will descend on Scottsdale, Arizona for PPMD’s 24thAnnual Conference! With our most expansive agenda to date, including presentations from dozens of researchers, clinicians, and industry partners, we wanted to make this year’s…

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