Blog

August 20, 2018

PPMD’s Certified Duchenne Care Center Program – August 2018 Bi-Annual Report

PPMD’s Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the CDC Care Considerations, available and accessible to as many families as possible, and to strengthen…

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Connect with other Duchenne Families in Your Area

Online interactions are great, but nothing can replace in-person connections with other families living with Duchenne. PPMD has local Connect groups all over the country for this very reason. Have you “connected” with your state’s PPMD Connect group…

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Continuing to Enhance The Duchenne Registry

Many of you have probably noticed the new “look and feel” of The Duchenne Registry (previously DuchenneConnect) – see the end of this blog for a list of some of the new features! In honor of our…

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Sarepta Therapeutics Announces Clinical Hold on Micro-Dystrophin Gene Therapy Trial

This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin Phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The company was notified by the Research…

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PPMD Hosts Largest Annual Conference — Presentations Available for Download!

Thank you to everyone who joined us in Scottsdale, AZ for the 2018 Annual Conference! While the extensive agenda, research presentations, break-out sessions, and interactions with industry leaders were all an incredible part of this…

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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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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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The Duchenne Carrier Characterization Study at Nationwide Children’s Hospital

May is a month to celebrate mothers. PPMD’s dedication to mothers in our community is, in small part, reflected in our dedication to doing all we can to care for female carriers of Duchenne. The…

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Center Directors Publish Collective Statement Regarding Patient Access to Approved Therapies

PPMD is proud to announce the publication of a collective statement regarding patient access to approved therapies from the center directors of our Certified Duchenne Care Centers. There are many ongoing treatment trials for Duchenne and…

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[WEBINAR] What is a Master Protocol?

PPMD fights to end Duchenne for every single family. As part of our mission, we believe that we must continually work to identify and explore innovative opportunities that may accelerate the drug development and clinical…

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