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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

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…

Learn More

January 12, 2026

PPMD’s Race to End Duchenne Team Member Claims Victories in Both 5K and 10K as Community Raises Funds for Duchenne and Becker Research and Care

PPMD celebrated a powerful and inspiring Walt Disney World® Marathon Weekend as part of its Race to End Duchenne endurance fundraising program this past weekend! PPMD runners, families, and supporters came together in Orlando, Florida,…

Learn More

December 22, 2025

Watch: Capricor Therapeutics — HOPE-3 Community Update (Webinar Recording)

PPMD recently held a community webinar with Capricor Therapeutics, during which the company shared their most recent results from the Phase 3 HOPE-3 trial evaluating deramiocel (CAP-1002), Capricor’s investigational cell therapy for the treatment of…

Learn More

December 16, 2025

Historic Victory: Duchenne Added to the Recommended Uniform Screening Panel (RUSP)

Today marks a monumental victory for the Duchenne community. The U.S. Department of Health and Human Services (HHS) has officially added Duchenne to the Recommended Uniform Screening Panel (RUSP)—the national list of conditions recommended for…

Learn More

December 8, 2025

Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne

Dyne Therapeutics has announced positive topline results from its Registrational Expansion Cohort (REC) of the Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in people living with Duchenne who are amenable…

Learn More