Blog

September 17, 2018

PPMD’s Ryan Fischer Joins the Muscular Dystrophy Coordinating Committee (MDCC)

When our community first came together with our Congressional champions in 1999 to carve a path forward for our Duchenne community, among the priorities we had was for all federal agencies that impacted our community…

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September 7, 2018

PPMD & Sarepta Partner to Launch Duchenne Outcomes Research Interchange

I am thrilled to announce PPMD’s plan to launch a patient- and physician-reported outcomes data warehouse – the Duchenne Outcomes Research Interchange (the Interchange) – which will combine data from The Duchenne Registry with clinician…

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September 5, 2018

PPMD Designates American Family Children’s Hospital a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named American Family Children’s Hospital in Madison, Wisconsin our 19th Certified Duchenne Care Center. PPMD’s Certified Duchenne Care Center Program, which supports standardized, comprehensive care and services for all people…

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August 30, 2018

Gene Repair Technology Restores Dystrophin Expression in Canine Model of Duchenne

We are encouraged to learn that Exonics Therapeutics, Inc., a biotechnology company focused on developing gene editing therapies to treat patients with Duchenne and other neuromuscular diseases, today announced the publication of a preclinical study…

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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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