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September 22, 2026

Catch up on PPMD Together: San Francisco

This past weekend, PPMD headed west for PPMD Together: San Francisco, CA, bringing together families, clinicians, industry partners, and local organizations for two days of connection, learning, and collaboration. Following an evening reception on Friday,…

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September 18, 2026

PPMD Celebrates Patient-Centered Advocacy at RAPS Convergence 2026

Earlier this week, PPMD joined global regulatory professionals, patient advocates, industry leaders, and others from across the healthcare community at RAPS Convergence 2026 in Charlotte, North Carolina. This year marked the 50th anniversary of the…

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September 8, 2026

Novartis Shares Del-Zota Regulatory Update: FDA Accepts BLA for Priority Review

Novartis announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the Biologics License Application (BLA) of del-zota (delpacibart zotadirsen) is sufficiently complete to permit a substantive review. According to…

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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 24, 2026

Precision Biosciences Doses First Patient in FUNCTION-DMD Trial of PBGENE-DMD

Precision BioSciences, Inc. today announced the dosing of the first patient in the Phase 1/2 FUNCTION-DMD clinical trial, evaluating the safety, tolerability, and efficacy of PBGENE-DMD for the treatment of Duchenne. PBGENE-DMD is Precision’s investigational…

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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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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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Ahead of the July 29 Advisory Committee: We Urge the FDA to Consider the Totality of Evidence

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…

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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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PPMD Honored with RAPS 2026 Patient-Centered Health Award

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…

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