June 21, 2023

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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June 20, 2023

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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June 19, 2023

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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June 16, 2023

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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June 15, 2023

PPMD, CureDuchenne, Muscular Dystrophy Association Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy

CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) today announced a collaborative clinical trial grant to test repurposing of the FDA-approved drug Vyvgart (developed by argenx), for its potential ability to reduce…

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June 13, 2023

Over 1,000 Free Carrier Tests Performed through PPMD’s Decode Duchenne Program!

We are thrilled to announce a new milestone achieved in the Decode Duchenne genetic testing program. We recently reached 1,000 carrier tests performed! This means that over 1,000 women have received free carrier testing through…

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June 8, 2023

Sarepta Announces Update on ENVISION Study Enrollment in U.S.

Today, Sarepta Therapeutics announced information regarding their ENVISION study for SRP-9001.  ENVISION is a 72-week placebo control design with crossover study of SRP-9001 (delandistrogene moxeparvovec) in older ambulatory and non-ambulatory individuals living with Duchenne muscular…

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June 7, 2023

FibroGen Announces Topline Data from Phase 3 LELANTOS-1 Study

This morning, FibroGen announced topline data from the Phase 3 LELANTOS-1 study of pamrevlumab, their investigational monoclonal antibody, for non-ambulatory patients with Duchenne muscular dystrophy.   Although generally safe and well tolerated, pamrevlumab did not…

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June 6, 2023

New Duchenne School Resources

Even though school is officially out for summer, it’s never too early to start planning for next year. We are here to help navigate this Duchenne journey together and connect you with any additional resources…

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June 2, 2023

Race Athlete Spotlight: Tiffany Mulloy

Tiffany Mulloy recently participated in the Cincinnati Flying Pig Marathon Weekend for the 2nd year in a row, in honor of her son Allan and all those living with Duchenne. Tiffany raised more than $11,000…

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