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PPMD Announces Pediatric Certified Duchenne Care Center at UPMC Children’s Hospital of Pittsburgh

Today Parent Project Muscular Dystrophy announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with the pediatric certification of the clinic at UPMC Children’s Hospital of Pittsburgh. Rachel Schrader, MS, APRN, CPNP-PC,…

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Edgewise Therapeutics Now Enrolling CANYON Phase 2 Clinical Trial Of EDG-5506 In Individuals With Becker Muscular Dystrophy

PPMD is excited to share that Edgewise Therapeutics has announced CANYON, their Phase 2 study of EDG-5506 in individuals with Becker, is now enrolling. EDG-5506 is a small molecule, taken orally, that is intended to…

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Dyne Therapeutics Announces FDA Clearance of IND Application for DYNE-251

Dyne Therapeutics has announced that the FDA has lifted the clinical hold and cleared its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in participants with Duchenne amenable to skipping exon…

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PPMD Announces Pediatric Certified Duchenne Care Center at St. Louis Children’s Hospital

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

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Sarepta Shares New Functional Data From SRP-9001 Duchenne Gene Therapy Clinical Development Program

Sarepta Therapeutics has shared new functional data demonstrating functional improvements across multiple studies from the clinical development program for SRP-9001 including one-year functional results compared to an external control group, from Study SRP-9001-103 (ENDEAVOR) in…

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Thank you for joining the Power of Us!

Thank you to everyone who joined the Race to End Duchenne Virtual Series – Power of Us, demonstrating the power of our community when we come together! Nearly 100 people joined the Power of Us…

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Race Athlete Spotlight: Sarah Finazzo

Joining the Race to end Duchenne team in 2022, Sarah has been off to a great start running and fundraising in honor of her nephew, Henry. Her first race with us was the NYC Half…

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PPMD Submits RUSP Nomination Package for Duchenne Muscular Dystrophy

PPMD’s Duchenne Newborn Screening efforts have reached an incredible milestone. Today, PPMD submitted a nomination package to add Duchenne to the Recommended Uniform Screening Panel (RUSP), requesting Duchenne be a condition recommended for nationwide Newborn…

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Santhera Updates on Ongoing NDA Submission for Vamorolone in Duchenne

Today Santhera shared an update on the timeline for the submission of their New Drug Application (NDA) for Vamorolone, an investigational dissociative steroid for individuals with Duchenne. Santhera expects to complete the submission of their…

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Capricor Announces Positive One-Year Results from HOPE-2 Open Label Extension Study Evaluating CAP-1002

Dr. Linda Marbán, CEO of Capricor Therapeutics, presented a Late-Breaking News session at PPMD’s 2022 Annual Conference, announcing positive 1-year efficacy results from the ongoing HOPE-2 open label extension study of CAP-1002 in non-ambulant Duchenne…

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