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Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53

Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals…

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Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne

Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts…

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Community Comes Together on Capitol Hill: A Recap of PPMD’s 2026 Advocacy Conference

Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with a shared mission: to ensure that the voices of individuals living with Duchenne and Becker and their families are heard…

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Capricor Therapeutics Announces Positive Data from HOPE-3 Study of Deramiocel

Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne. Deramiocel is the company’s investigational cell therapy for the treatment of Duchenne cardiomyopathy….

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Solid Biosciences Shares Interim Positive Update on Phase 1/2 INSPIRE DUCHENNE Trial

Solid Biosciences Inc. has shared updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin…

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REGENXBIO Shares Positive Interim Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202

REGENXBIO has announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via…

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Edgewise Announces Positive Long-Term Sevasemten Data

Edgewise Therapeutics, Inc. has shared long-term data from its MESA open-label extension study of sevasemten in Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in…

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Capricor Therapeutics Announces New PDUFA Date for Deramiocel

Capricor Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has lifted the July 2025 Complete Response Letter and resumed review of the company’s Biologics License Application (BLA) for Deramiocel, an investigational cell…

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PPMD and FED Announce 2026 Advocacy Leadership Awards Honoring Bipartisan Champions in Congress

PPMD, in partnership with the Foundation to Eradicate Duchenne (FED), is pleased to announce Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy Klobuchar (MN), Representative Doris Matsui (CA-07), and Senator Roger Wicker (MS)…

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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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