December 15, 2023

Wave Life Sciences Announces Initiation of Dosing in Phase 2 Duchenne Clinical Trial of WVE-N531

Wave Life Sciences recently shared that the company has initiated dosing and has fully enrolled the Phase 2 FORWARD-53 trial of investigational WVE-N531, a potential exon skipping therapy targeting people with Duchenne amenable to exon…

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December 12, 2023

Watch: Community Update on the New INSPIRE DUCHENNE Clinical Study of SGT-003 (Webinar Recording)

Solid Biosciences recently joined PPMD for a community webinar to provide an overview of INSPIRE DUCHENNE, a first-in-human clinical study evaluating SGT-003, a next-generation microdystrophin gene therapy candidate for the treatment of Duchenne. The Solid…

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December 5, 2023

PTC Therapeutics Shares Update on Translarna™ Regulatory Activities

PTC Therapeutics, Inc. has shared an update on regulatory activities in Europe and the United States for Translarna™ (ataluren), a treatment for Duchenne patients with nonsense variants.  Europe: European Medicines Agency (EMA) Update Following the…

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November 14, 2023

Solid Biosciences Receives FDA Clearance for Investigational New Drug Application for SGT-003 Gene Therapy Candidate

PPMD is pleased to learn that Solid Biosciences has received clearance by the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application for SGT-003, the company’s next generation microdystrophin gene therapy…

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November 7, 2023

PPMD Provides $250,000 in Funding to Rare Disease Research to Establish New Clinical Trial Site

PPMD has provided $250,000 in funding to Rare Disease Research, LLC (RDR), to open a second independent rare disease clinical research site in the Raleigh-Durham metropolitan area in North Carolina. With this funding commitment, we…

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October 30, 2023

Sarepta Therapeutics Announces Topline Results from Phase 3 EMBARK Study of ELEVIDYS Gene Therapy

Sarepta Therapeutics has announced Phase 3 topline results of their EMBARK study with ELEVIDYS, a micro-dystrophin gene therapy product currently approved for patients aged 4-5 living with Duchenne. Sarepta shares that while EMBARK did not…

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October 30, 2023

WATCH: Capricor Therapeutics – CAP-1002 HOPE-3 Program Update (Webinar Recording)

PPMD and Capricor Therapeutics held a community webinar on October 25, 2023 to provide an overview of the latest regulatory developments for Capricor’s ongoing HOPE-3 Phase 3 study of CAP-1002, the company’s cell therapy. Capricor’s…

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October 27, 2023

Edgewise Therapeutics Announces Expansion of their EDG-5506 Clinical Program in Duchenne

Edgewise Therapeutics, Inc. has announced an expansion of their clinical development program of EDG-5506.  EDG-5506, which comes in the form of a small pill, is an investigational product that aims to protect muscles, specifically fast-twitch…

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October 26, 2023

FDA Grants Approval to AGAMREE® (Vamorolone)

PPMD is excited to learn that the FDA has granted approval to AGAMREE® (vamorolone), a dissociative steroid indicated for individuals diagnosed with Duchenne muscular dystrophy from age 2 years and older.  AGAMREE® is a first-in-class…

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October 24, 2023

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

Edgewise Therapeutics joined PPMD for a community webinar on October 18, 2023 to discuss the company’s investigational compound, EDG-5506. Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer at Edgewise, and Alan Russell, Ph.D., Edgewise’s Co-Founder and…

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