November 21, 2024

WATCH: RGX-202 – AFFINITY DUCHENNE Pivotal Program and Functional Data (Webinar Recording)

REGENXBIO recently joined PPMD for a community webinar to provide an update on its AFFINITY DUCHENNE trial of RGX-202, REGENXBIO’s microdystrophin gene therapy. Dr. Naz Dastgir discussed the company’s pivotal trial plans and shared interim…

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November 5, 2024

WATCH: Exploring Muscle Regeneration – A New Approach with Satellos (Webinar Recording)

Satellos recently joined PPMD for a community webinar exploring a new approach for the treatment of Duchenne. Satellos presented on the discovery behind SAT-3247, a small molecule drug that mobilizes the body’s own muscle stem…

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October 11, 2024

WATCH: SHIELD DMD – A Bone Health Study in Duchenne (Webinar Recording)

Bone health is an important aspect of living with Duchenne muscular dystrophy. Roche recently joined PPMD for a webinar during which Maitea Guridi, a Principal Clinical Scientist at Roche, shared learnings around the natural history…

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October 3, 2024

WATCH: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy (Webinar Recording)

Wave Life Sciences recently joined PPMD for a presentation of interim results from the company’s ongoing Phase 2 FORWARD-53 study of WVE-N531 in individuals living with Duchenne muscular dystrophy who are amenable to exon 53…

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September 19, 2024

PPMD Community Webinar Recap: Overview of DUVYZAT and Access Resources

On September 18, 2024, PPMD hosted an informative community webinar featuring representatives from ITF Therapeutics, PPMD, and the Little Hercules Foundation. The session focused on DUVYZAT (givinostat), a recently approved HDAC inhibitor for the treatment…

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August 20, 2024

WATCH: Advancing DYNE-251 – An Investigational Medicine for Duchenne to Deliver for Patients (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a presentation of clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping, first released on May…

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August 9, 2024

WATCH: Edgewise Therapeutics – Clinical Trials in Becker Muscular Dystrophy (Webinar)

Edgewise Therapeutics recently joined PPMD for a webinar to provide the Becker community information about the company’s clinical trials for Becker. Edgewise’s Chief Scientific Officer and Chief Medical Officer, shared two year results from the…

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May 6, 2024

WATCH: Community Progress in Duchenne Newborn Screening (Webinar Recording)

PPMD, in collaboration with the Muscular Dystrophy Association (MDA), the EveryLife Foundation for Rare Diseases, and the Little Hercules Foundation (LHF), recently hosted a webinar on the background, history, and next steps for Duchenne Newborn…

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March 28, 2024

WATCH: Community Update with ITF Therapeutics – Introducing DUVYZAT™ (givinostat) (Webinar Recording)

ITF Therapeutics recently joined PPMD for a webinar to provide the Duchenne community with an opportunity to meet members of the ITF Therapeutics team to learn more about their organization and next steps to support…

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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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