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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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Exercise in Dystrophinopathy: What Families Need to Know

PPMD recently hosted a webinar centered on common questions from families: is exercise recommended in dystrophinopathy care and how it can be done safely? Experts discussed what we currently understand, what researchers are still exploring,…

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Momentum Continues: Duchenne Newborn Screening Advances Across the US

Duchenne was added to the Recommended Uniform Screening Panel (RUSP) (RUSP) on December 16, 2025, and in the months since, that decision, coupled with our continued advocacy efforts, have driven meaningful action at the state…

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