Blog

October 22, 2026

Becker Education and Engagement Day 2026: Building Knowledge, Connection, and Community

A guest blog authored by Nevin Steiner, PPMD’s Adult Advisory Committee (PAAC) Becker Officer Living with Becker muscular dystrophy looks different for everyone. Our experiences, symptoms, care needs, and goals may vary, but we share…

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October 15, 2026

Care Should Grow With You: Building a Better Future for Adults With Duchenne and Becker

For families living with Duchenne and Becker, planning for adulthood should bring possibilities: education, relationships, work, greater independence, and the opportunity to build a life shaped by individual goals and choices. Too often, it also…

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October 5, 2026

Stay Healthy This Respiratory Season: What to Know and How to Prepare

As school gets underway and winter approaches, respiratory viruses like influenza (flu), RSV, and COVID-19 are more likely to spread. This year, and every year, it’s important to protect yourself, your children, and your family…

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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 21, 2026

Deramiocel: Where We Are Today

As we approach August 22, the FDA’s current Prescription Drug User Fee Act (PDUFA) action date for Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, PPMD recognizes how closely the Duchenne community is watching for…

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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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Thank You for Raising Your Voices During the Deramiocel FDA Advisory Committee Meeting

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…

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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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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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Massachusetts Begins Newborn Screening for Duchenne

As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor…

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