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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... The post Update on the FDA’s Review of Deramiocel: Continuing the Path Forward appeared first on Parent Project Muscular Dystrophy…
Today, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met to discuss Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne muscular dystrophy (Duchenne). Following a full day... The post Thank You for Raising Your Voices During the Deramiocel FDA Advisory Committee Meeting appeared first on Parent Project…
On July 29th, 2026, the FDA will convene an Advisory Committee meeting to evaluate Deramiocel, Capricor Therapeutics’ investigational therapy for Duchenne muscular dystrophy. These meetings are an important step in the regulatory process, bringing together... The post Ahead of the July 29 Advisory Committee: We Urge the FDA to Consider the Totality of Evidence appeared first on Parent Project…
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... The post What to Know Ahead of This Week’s Deramiocel FDA Advisory Committee Meeting appeared first on Parent Project Muscular Dystrophy .
PPMD is honored to receive the 2026 Patient-Centered Health Award from the Regulatory Affairs Professionals Society (RAPS), recognizing organizations that have made significant contributions to advancing patient-centered healthcare and ensuring patient perspectives inform the development... The post PPMD Honored with RAPS 2026 Patient-Centered Health Award appeared first on Parent Project Muscular…
Today, Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) for the treatment of individuals living with Duchenne muscular dystrophy who... The post FDA Accepts Dyne Therapeutics’ Biologics License Application for Z-Rostudirsen appeared first on Parent Project Muscular…
Whether you joined us in Orlando or are catching up from home, the learning opportunities from PPMD’s 2026 Annual Conference continue. We’re excited to share that recordings and resources from PPMD’s 2026 Annual Conference are... The post Highlights from PPMD’s 2026 Annual Conference appeared first on Parent Project Muscular Dystrophy .
The FDA has scheduled an Advisory Committee meeting for July 29, 2026 to review Deramiocel, Capricor’s investigational cell therapy for Duchenne muscular dystrophy. The meeting will be held in person with the option to participate... The post Preparing for the Deramiocel FDA Advisory Committee Meeting: How to Submit Written Testimony appeared first on Parent Project Muscular Dystrophy .
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... The post FDA Accepts sNDAs to Review Traditional Approval for AMONDYS 45 and VYONDYS 53 appeared first on Parent Project Muscular Dystrophy…
PPMD’s 2026 Annual Conference brought together individuals and families from across the Duchenne and Becker community for three days filled with scientific presentations, discussions around care, clinical trials and quality of life, and of course,... The post Catching Up on PPMD’s 2026 Annual Conference: Part II appeared first on Parent Project Muscular Dystrophy .