March 15, 2024

WATCH: NS Pharma – Overview of New Exon Skipping Trials: Brogidirsen and NS-050/NCNP-03 (Webinar Recording)

NS Pharma recently joined PPMD for a community webinar to share an overview of two of the company’s new exon skipping trials: Brogidirsen (exon 44) and NS-050/NCNP-03 (exon 50). Vamshi Rao, MD, discussed these two…

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February 23, 2024

Watch: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences (Webinar Recording)

Wave Life Sciences recently joined PPMD for a community webinar to share an update on the company’s FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular…

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February 14, 2024

WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)

PPMD was recently joined by the team at PTC Therapeutics to discuss upcoming changes and continuing community support around EMFLAZA (deflazacort). EMFLAZA came on the U.S. market in 2017 as the first FDA-approved steroid specifically…

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February 8, 2024

Watch: Dyne Therapeutics — Advancing DYNE-251, an Investigational Medicine for DMD (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a community webinar to present initial clinical data from the DELIVER trial of DYNE-251, a next-generation exon skipping investigational product, in individuals with Duchenne who are 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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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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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

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 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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October 17, 2023

WATCH: Supporting Families Through Decision Making (Webinar Recording)

PPMD webinar explores how palliative care teams can support families through decision making PPMD’s Alexis Hazlett and Patrick Moeschen were recently joined by panelists Dr. Lauren Treat, Neurologist and Palliative Care Physician at Colorado Children’s,…

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