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 29, 2023

Italfarmaco Announces PDUFA Extension for the FDA’s Review of Givinostat

Italfarmaco Group announced an extension in the FDA’s review of the New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the potential treatment of Duchenne. The new Prescription Drug User…

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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 14, 2023

Advancing Duchenne Cardiac Care: A Year of Impact and Gratitude

Cardiac care is at the heart of Duchenne, which is why it remains a critical element of PPMD’s efforts to optimize care and research through our ongoing Cardiac Initiative. As we look back on the…

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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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November 3, 2023

Defeat Duchenne Canada and PPMD Award $300,000 (USD) Clinical Fellowship in Duchenne Endocrinology and Bone Fragility

PPMD and Defeat Duchenne Canada have announced a collaborative research award of $300,000 (USD) in support of two Clinical Fellowships in Duchenne Endocrinology and Bone Fragility. The three-year award will sponsor the extended fellowship of…

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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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