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Over 1,000 Free Carrier Tests Performed through PPMD’s Decode Duchenne Program!

We are thrilled to announce a new milestone achieved in the Decode Duchenne genetic testing program. We recently reached 1,000 carrier tests performed! This means that over 1,000 women have received free carrier testing through…

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Sarepta Announces Update on ENVISION Study Enrollment in U.S.

Today, Sarepta Therapeutics announced information regarding their ENVISION study for SRP-9001. ENVISION is a 72-week placebo control design with crossover study of SRP-9001 (delandistrogene moxeparvovec) in older ambulatory and non-ambulatory individuals living with Duchenne muscular…

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FibroGen Announces Topline Data from Phase 3 LELANTOS-1 Study

This morning, FibroGen announced topline data from the Phase 3 LELANTOS-1 study of pamrevlumab, their investigational monoclonal antibody, for non-ambulatory patients with Duchenne muscular dystrophy. Although generally safe and well tolerated, pamrevlumab did not…

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Sarepta Announces PDUFA Extension and Update for the FDA’s Review of SRP-9001

Sarepta Therapeutics announced today that they received an update from the FDA on the Biologics License Application (BLA) for SRP-9001. The PDUFA date reportedly has been extended to June 22, 2023. …

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A Community United: Thank You for Raising Your Voice During the SRP-9001 Advisory Committee

Today was a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne. This meeting was held as part of the regulatory review process for…

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WATCH: Edgewise — A Novel Approach Designed to Protect Muscle in Becker (Webinar Recording)

PPMD was joined by Edgewise Therapeutics for a community webinar on May 3, 2023 to discuss the company’s novel approach designed to protect muscle in individuals living with Becker muscular dystrophy. The webinar highlighted EDG-5506,…

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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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