Blog

Join our 2023 Coach to Cure MD team!

Coach to Cure MD returns September 30, 2023! This year marks the 16th year of Coach to Cure MD! We need you and your family to join our team as we rally together on Saturday,…

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Duchenne Newborn Screening milestone—first state with universal newborn screening for Duchenne!

For more than a decade, PPMD has worked towards newborn screening (NBS) for Duchenne, including initiating a pilot study in New York State and developing a nomination package for federal review as a recommended condition…

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Catch up on what’s happening at PPMD’s 2023 Annual Conference: Day 3

The third and final day of PPMD’s 2023 Annual Conference kicked off with the much-anticipated Gene Therapy Town Hall. Buddy Cassidy opened the session by sharing his experience as a Patient Representative for the May…

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Catch Up on What’s Happening at PPMD’s 2023 Annual Conference: Day 2

If you are here at PPMD’s Annual Conference this year, you’ve likely seen a man with a camera running around snapping pictures. Rick Guidotti, photographer and Founder & CEO of Positive Exposure, delivered keynote remarks…

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Catch Up on What’s Happening at PPMD’s 2023 Annual Conference: Day 1

This year’s Annual Conference brings together more than 1,000 families, researchers, clinicians, and industry partners from around the world for three days of informative and interactive sessions featuring the latest news in research, clinical trials,…

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Italfarmaco Group Announces NDA Accepted for Givinostat

PPMD is excited to learn that the FDA completed its filing review and accepted Italfarmaco Group’s New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the treatment of individuals with…

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Edgewise Therapeutics announces positive topline 12-month data from EDG-5506 ARCH study

Edgewise Therapeutics today announced topline 12-month data from the ARCH study of EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in patients with Becker muscular dystrophy. The ARCH study is evaluating…

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FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy…

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Santhera announces exclusive North America license agreement with Catalyst Pharmaceuticals for vamorolone

PPMD has learned that Santhera Pharmaceuticals has entered into an agreement granting exclusive North America license for vamorolone to Catalyst Pharmaceuticals. Santhera will continue to keep commercialization rights in Europe, while Catalyst will obtain commercialization…

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Thanks to you PPMD continues forward in all areas of support

Thanks to the commitment and generosity of each of you, Parent Project Muscular Dystrophy will invest more than $13 million this year into key programs and strategies to continue to effectively move the Duchenne landscape…

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