November 6, 2026

Satellos Announces Change to Primary Endpoint in BASECAMP Duchenne Trial

Satellos Bioscience has announced a change to the primary endpoint in its Phase 2 BASECAMP clinical trial evaluating forazapadin, an investigational oral, small molecule designed to enhance skeletal muscle repair and regeneration in individuals living…

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November 5, 2026

Sarepta Shares Update on ENVISION Clinical Trial and Ongoing Duchenne Research

Today, Sarepta Therapeutics shared an update through a community letter regarding its research and clinical development programs, including an important decision about the Phase 3 ENVISION clinical trial evaluating ELEVIDYS in non-ambulatory and late-ambulatory individuals…

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November 4, 2026

Servier Announces Topline Results from Pivotal Sevasemten Study in Becker

Servier announced yesterday that the pivotal Phase 3 GRAND CANYON study evaluating sevasemten in individuals living with Becker muscular dystrophy did not meet its primary endpoint. Sevasemten is an orally administered small molecule inhibitor designed…

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October 22, 2026

Fellowship Update: Universal Antibody Screening Test for AAV Gene Therapy

As gene therapy research continues to advance, understanding who may be eligible for a particular treatment remains an important challenge for the Duchenne and Becker community. PPMD is committed to supporting research that addresses these…

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September 30, 2026

Catch up on the 31st Congress of the World Muscle Society

Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. The meeting offers a dynamic platform for sharing new data, fostering collaborations,…

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September 8, 2026

Novartis Shares Del-Zota Regulatory Update: FDA Accepts BLA for Priority Review

Novartis announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the Biologics License Application (BLA) of del-zota (delpacibart zotadirsen) is sufficiently complete to permit a substantive review. According to…

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August 24, 2026

Update on Deramiocel: PDUFA Date Extended

Today, Capricor Therapeutics formally announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s amendment to the Biologics License Application (BLA) for Deramiocel, and the FDA will be extending the current Prescription…

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August 24, 2026

Precision Biosciences Doses First Patient in FUNCTION-DMD Trial of PBGENE-DMD

Precision BioSciences, Inc. today announced the dosing of the first patient in the Phase 1/2 FUNCTION-DMD clinical trial, evaluating the safety, tolerability, and efficacy of PBGENE-DMD for the treatment of Duchenne. PBGENE-DMD is Precision’s investigational…

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August 13, 2026

Update on the FDA’s Review of Deramiocel: Continuing the Path Forward

Today, Capricor Therapeutics shared an important update on the FDA’s review of the Biologics License Application (BLA) for Deramiocel, the company’s cell therapy for the treatment of Duchenne muscular dystrophy-associated cardiomyopathy. Capricor has indicated that…

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July 27, 2026

What to Know Ahead of This Week’s Deramiocel FDA Advisory Committee Meeting

On Wednesday, July 29, 2026, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) will meet to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne. The meeting…

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