September 12, 2018

20th Annual Mitchell’s Run Thru Rockford Another Huge Success to Help End Duchenne

On August 18th, the 20th Annual Mitchell’s Run Thru Rockford raised an incredible $85,000 to help our fight to end Duchenne. This was the most successful event to date! Thank you to Steve, Sandy, Halle,…

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September 11, 2018

Natural disaster resources for families living with Duchenne

We are officially in the midst of hurricane season, with a major storm aimed at a large portion of the east coast later this week. To all of our PPMD Family in the path of…

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September 10, 2018

VIDEO: Head, Shoulders, Knees, and Toes

There are simple things we know will help our children living with Duchenne. But with all of the big things that we try to remember to do every day (and often several times a day!),…

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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 6, 2018

Updated Duchenne Care Guide for Families

You have probably heard a lot about ‘Care Considerations’. These were the first Standards of Care published in 2010. The purpose of developing Standards of Care is to ensure every person living with the diagnosis…

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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 30, 2018

Pfizer Terminates Domagrozumab (PF-06252616) Clinical Studies for the Treatment of Duchenne

We are deeply disappointed to learn that Pfizer is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne: a Phase 2 safety and efficacy study (B5161002) and an open-label extension study…

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August 29, 2018

One Month Till Game Day!

It’s the start of the football season and we want YOU to help your local team celebrate this year’s 11th Annual Coach To Cure MD! There’s still plenty of time for you to put together…

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August 23, 2018

Overview: Access to Unapproved Investigational Drugs in the U.S.

Overview: Access to Unapproved Investigational Drugs in the U.S.* Like you, PPMD has had many questions about the way we can access UNAPPROVED investigational therapies in Duchenne. Expanded access, compassionate use, Right to Try –…

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