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May 12: Advisory Committee Meeting for SRP-9001 – Live Stream Information & Meeting Materials Released

The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’s investigational gene therapy, which has applied for FDA approval through the Accelerated Approval pathway with…

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What You Need To Know Ahead of the SRP-9001 Advisory Committee on Friday, May 12

This Friday, May 12th, will be a historic day for the Duchenne community. It will be the day of the first ever FDA Advisory Committee meeting for a gene therapy specifically to treat Duchenne. PPMD…

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PPMD Submits Findings from New Gene Therapy Preference Study to FDA for SRP-9001 Advisory Committee

May 12 will be a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy specifically intended to treat Duchenne. PPMD, our community partners, and many others have long…

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PPMD & Community Partners Submit Letter to FDA Encouraging Appropriate Regulatory Flexibility in SRP-9001 Review

PPMD along with community partners and key opinion leaders and physicians in the Duchenne space have submitted a letter to the US Food and Drug Administration (FDA) and FDA’s Center for Biologics Evaluation and Research…

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Pfizer Completes Enrollment of CIFFERO Phase 3 and DAYLIGHT Phase 2 Gene Therapy Trials

Pfizer announced today that enrollment is now complete for both the Phase 3 CIFFERO study and Phase 2 DAYLIGHT study of PF-06939926 (fordadistrogene movaparvovec) in Duchenne. As always, we are so grateful to all of…

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WATCH: Dyne Therapeutics — Advancing FORCE in the Pursuit of Therapies (Webinar Recording)

Dyne Therapeutics joined PPMD for a community webinar on April 12, 2023 to discuss Dyne’s FORCE™ platform. Ashish Dugar, PhD, MBA, Dyne’s Chief Medical Affairs Officer, and Molly White, Dyne’s Vice President, Global Head of…

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How to Submit Written Testimony for the SRP-9001 FDA Advisory Committee Meeting – Deadline May 5th

Sarepta recently announced that the FDA Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12th. The event will be completely virtual and a…

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PPMD’s Certified Duchenne Care Center Program – 2022 Impact & Progress

Every single person living with Duchenne deserves the best care possible. It is important that families know who is in charge of delivering the care they need and understand what centers are providing optimal, standardized…

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NS Pharma Announces FDA Clearance of IND for Clinical Trial of NS-089/NCNP-02 Exon 44 Skipping Candidate

PPMD is excited to share that NS Pharma has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for NS-089/NCNP-02, a potential exon skipping therapy targeting people with…

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Sarepta Announces Date for SRP-9001 FDA Advisory Committee Meeting: Make Your Voice Heard on May 12th

Today Sarepta shared that the FDA has announced that the planned Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12, 2023. The event…

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