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Living with a rare disease is incredibly complex, with countless layers to navigate. Managing daily life amid so many unknowns — and trying to find a regimen that truly works — can feel overwhelming. From the routine of medications and other relentless therapies to countless medical frustrations, we’re forced to become experts in our own…

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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 therapy for the treatment of Duchenne cardiomyopathy. The FDA has assigned a new Prescription Drug User Fee Act (PDUFA) target…

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At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases. Muscular Dystrophy News Today sat down with John F. Crowley, president and CEO of the Biotechnology Innovation Organization (BIO) and this…

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As an early funder of Capricor Therapeutics, CureDuchenne is pleased to share that the FDA has set the PDUFA target action date for August 22, 2026 as the deadline to review approval of deramiocel for Duchenne muscular dystrophy.  Deramiocel is a cell therapy that exerts immunomodulatory and anti-fibrotic actions on cardiac and skeletal muscle.   Read…

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Edgewise Therapeutics, a CureDuchenne funded company, today announced positive long-term Sevasemten data that demonstrated sustained functional stabilization in Becker Muscular Dystrophy (BMD) patients through 3.5 years of treatment. This result is in stark contrast to the functional decline expected from BMD natural history data. Today’s result is particularly important for BMD patients, an underserved population,…

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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 muscular dystrophies. MESA is an open-label extension evaluating sevasemten long-term safety, tolerability, and efficacy in adults and adolescents with Becker…

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Sharon Hesterlee, PhD, MDA President and CEO On March 9, Sharon Hesterlee, PhD, MDA President and CEO, welcomed more than 2,400 people attending MDA’s annual Clinical & Scientific Conference, both in person at the Hilton Orlando in Florida and virtually. The conference is the largest global gathering of neuromuscular clinicians, researchers, industry partners, advocacy organizations,…

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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) as recipients of the 2026 Advocacy Leadership Awards. The awards honor bipartisan leaders in Congress who have demonstrated exceptional commitment…

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I recently found myself rummaging through a very old collection of things from my childhood that my mother had preserved. Raise your hand if you have stuff like this: a box of certificates and trophies that you don’t remember, newspaper clippings of school events, scouting programs, a math paper from elementary school, and some sort…

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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 advancing critical policies that are transforming the lives of those affected by Duchenne and Becker muscular dystrophy. These accomplishments reflect…

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