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Edgewise Therapeutics announces positive topline 12-month data from EDG-5506 ARCH study

Edgewise Therapeutics today announced topline 12-month data from the ARCH study of EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in patients with Becker muscular dystrophy. The ARCH study is evaluating…

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Santhera announces exclusive North America license agreement with Catalyst Pharmaceuticals for vamorolone

PPMD has learned that Santhera Pharmaceuticals has entered into an agreement granting exclusive North America license for vamorolone to Catalyst Pharmaceuticals. Santhera will continue to keep commercialization rights in Europe, while Catalyst will obtain commercialization…

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Sarepta webinar recording

WATCH: ENVISION Study Update With Sarepta Therapeutics (Webinar Recording)

Last week Sarepta Therapeutics joined PPMD to discuss ENVISION, their upcoming 18-month placebo-controlled Phase 3 study of SRP-9001 (delandistrogene moxeparvovec) for older ambulatory and non-ambulatory individuals with Duchenne. Matthew Furgerson, PhD, Medical Director, Clinical Development…

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PPMD, CureDuchenne, Muscular Dystrophy Association Announce Collaborative Project to Focus on Re-Dosing Gene Therapy in Duchenne Muscular Dystrophy

CureDuchenne, Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) today announced a collaborative clinical trial grant to test repurposing of the FDA-approved drug Vyvgart (developed by argenx), for its potential ability to reduce…

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Female patient sits with doctor

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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