Blog

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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PAAC Travel Tips

Anyone living with Duchenne muscular dystrophy knows that there are extra steps to EVERYTHING. Traveling with medical equipment requires planning and organization. PPMD’s Adult Advisory Committee (PAAC) has put together several resources with tips that…

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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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Sarepta webinar recording

WATCH: ENVISION Study Update With Sarepta Therapeutics (Webinar Recording)

Last week Sarepta Therapeutics joined PPMD to discuss ENVISION, their upcoming 18-month placebo-controlled Phase 3 study of SRP-9001 (delandistrogene moxeparvovec) for older ambulatory and non-ambulatory individuals with Duchenne. Matthew Furgerson, PhD, Medical Director, Clinical Development…

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Norton Children's Hospital CDCC

PPMD Announces Pediatric Certified Duchenne Care Center at Norton Children’s Hospital

Today Parent Project Muscular Dystrophy announced the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Norton Children’s in Louisville, KY. This is an exciting step for…

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Parents meet with doctor

PPMD publishes results of study to understand Duchenne patients’ and caregivers’ perspectives on corticosteroids

Earlier this month, the publication, A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy, was accepted into the Journal of Neuromuscular Diseases. Background PPMD has led…

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PPMD Announces Pediatric Certified Duchenne Care Center at Boston Children’s Hospital

Today Parent Project Muscular Dystrophy announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with the certification of Boston Children’s Hospital. This is an exciting step for the CDCC Program as it…

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