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 13, 2023

WATCH: Overview of Topline Results from Phase 3 EMBARK Study (Webinar recording)

PPMD recently held a community webinar to discuss the recently shared Phase 3 topline results of the EMBARK study (SRP-9001-301). Crystal Proud, MD, provided an overview of the study topline results, highlighting that EMBARK achieved…

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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 6, 2023

WATCH: AGAMREE Approval – What this means for you & your family (Webinar recording)

PPMD was recently joined by Dr. Aravindhan Veerapandiyan of Arkansas Children’s Hospital to discuss the FDA’s recent approval of AGAMREE (vamorolone), a new steroid approved for the treatment of Duchenne muscular dystrophy in individuals aged…

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November 4, 2023

Calling All Adults with Duchenne & Becker: Join the 2024 PPMD Adult Advisory Committee (PAAC)

Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy…

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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 31, 2023

WATCH: Advocating for Access – Overview of Medicaid DUR and P&T Meetings (Webinar Recording)

During October’s Advocating for Access webinar, the PPMD and Little Hercules Foundation teams dug into what happens after a drug is approved with regard to how state Drug Utilization Review (DUR) boards assess how a…

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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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