March 9, 2026

Advocates Unite in Washington to Advance Duchenne and Becker Policy, Mark 25th Anniversary of MD-CARE Act

PPMD heads to Capitol Hill today with a group of more than 120 Duchenne and Becker advocates to meet with Congressional leaders during this year’s annual PPMD Advocacy Conference. “Together, our community has secured victories…

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March 6, 2026

PPMD Partners with Cooperative International Neuromuscular Research Group, Global Sponsors to Advance Expanded Duchenne Natural History Study

PPMD is proud to share our partnership with the Cooperative International Neuromuscular Research Group (CINRG) to advance the expanded Duchenne Natural History Study (eDNHS) study. Joining PPMD in support of the eDNHS study is Insmed…

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February 26, 2026

25 Years Later: Reflecting on the 2001 MD-CARE Act Hearing and Where We Are Now

On Thursday, February 26, advocates, clinicians, patient leaders, and policymakers came together for a powerful Senate Special Committee on Aging hearing titled “From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” This conversation served as…

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February 20, 2026

Roche Announces Termination of Phase 2 SHIELD DMD Study

We are disappointed to learn the news that Roche has made the difficult decision to terminate their Phase 2 SHIELD DMD study evaluating satralizumab for bone health in Duchenne.  According to Roche, this decision was…

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February 12, 2026

PTC Therapeutics Provides Regulatory Update on Translarna™ (atalauren)

My heart is heavy today following PTC’s decision to withdraw their NDA submission for atalauren (Translarna). PTC submitted their package to FDA in 2024, based on FDA’s commitment to review the totality of evidence for…

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February 11, 2026

Rare Disease Advocacy Delivers: Major Federal Wins for Duchenne and Becker Community

We applaud a major legislative victory for the rare disease and Duchenne and Becker community — one made possible because advocates like you raised your voices, shared your stories, and sustained pressure on lawmakers. With…

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February 6, 2026

Cardiac Care for Female Carriers

While February may be known for Valentine’s Day candy and flowers, it is also American Heart Month!  Within the dystrophinopathy community, keeping heart health front and center is especially critical. It is estimated that around…

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January 27, 2026

PPMD Joins Collaborative Research Effort to Decode How Human Muscle Regenerates

PPMD is proud to partner with the Muscular Dystrophy Association (MDA), the FSHD Society, and the LGMD2L Foundation on a new, collaborative research grant focused on one of the most fundamental — and still unanswered…

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January 26, 2026

Sarepta Shares Topline Three-Year EMBARK Data for ELEVIDYS in Ambulatory Duchenne Patients

Sarepta Therapeutics, Inc. has announced positive topline three-year functional results from Part 1-treated patients in EMBARK, the company’s global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals living with Duchenne. According…

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January 23, 2026

Solid Biosciences Shares Update on INSPIRE DUCHENNE Trial Evaluating SGT-003

Solid Biosciences, Inc., has shared updates on the company’s gene therapy program for the treatment of Duchenne. According to Solid, 33 participants have now been dosed in the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial…

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