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FDA Accepts sNDAs to Review Traditional Approval for AMONDYS 45 and VYONDYS 53

Today, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Applications (sNDAs) seeking traditional approval for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen). The FDA will…

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Solid Biosciences Shares Updates on SGT-003

Solid Biosciences has shared a new letter to the Duchenne community providing updates on the development of SGT-003, the company’s investigational gene therapy for Duchenne. Solid shared that the ongoing INSPIRE DUCHENNE Phase 1/2 clinical…

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Capricor Therapeutics Shares Positive Five-Year HOPE-2 OLE Data for Deramiocel in Duchenne

Today, Capricor Therapeutics announced positive five-year data from the company’s ongoing HOPE-2 open label extension (OLE) study of Deramiocel, Capricor’s novel cell therapy, in individuals living with Duchenne. According to Capricor, among the nine patients…

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Inside the HHS Clinical Trials Roundtable: Why Speed, Access, and Rigor Matter for the Duchenne Community

On June 22, 2026, PPMD Chief Executive Officer Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and…

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Dyne Submits BLA to FDA for z-rostudirsen (DYNE-251) in Duchenne Amenable to Exon 51 Skipping

Today, Dyne Therapeutics announced that the company has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zeleciment rostudirsen (z-rostudirsen, DYNE-251) for individuals living with Duchenne who are amenable…

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Dyne Announces Phase 3 Trial of Z-Rostudirsen, Plans for Submitting Biologics License Application to FDA

Dyne Therapeutics today announced the initiation of the company’s Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, DYNE-251), in individuals living with Duchenne who are amenable to exon 51 skipping. Z-rostudiresen is an investigational exon…

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GEMMABio Announces Duchenne Muscular Dystrophy Program, Shares Preclinical Data for Investigational Gene Therapy for Duchenne

Today, Gemma Biotherapeutics (GEMMABio), a clinical‑stage, global, genetic therapeutics company, shared information about the company’s Duchenne muscular dystrophy program, presenting preclinical data supporting candidate declaration for GB703, a novel, investigational gene therapy for the treatment…

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REGENXBIO Shares Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202

REGENXBIO Inc. has announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne…

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Solid Biosciences Doses First Participant in Phase 3 IMPACT DUCHENNE Clinical Trial Evaluating SGT-003 in Duchenne

Solid Biosciences Inc. has shared that the first participant has been dosed in IMPACT DUCHENNE, the company’s multi-country, placebo-controlled, randomized, double-blind, Phase 3 clinical trial investigating SGT-003 for the treatment of Duchenne muscular dystrophy. SGT-003…

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Entrada Reports Positive Topline Results from Phase 1/2 ELEVATE-44-201 Study in Duchenne

Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular…

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