Blog

Watch: Dyne Therapeutics — The Clinical Impact of the FORCE™ Platform for Duchenne (Webinar Recording)

Dyne Therapeutics recently joined PPMD for a community webinar on the DELIVER clinical trial evaluating DYNE-251. The Dyne team provided the latest updates on the Phase 1/2 global DELIVER study of the company’s investigational therapy…

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Steroids and Stress Dosing: Understanding the Updated PJ Nicholoff Steroid Protocol

Last week, PPMD brought together leading experts to address steroids, stress dosing and the updated PJ Nicholoff Steroid Protocol. Drs. Aravindhan Veerapandiyan, David Weber, and Anne Marie Sbrocchi shared valuable insights on the management of…

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Watch: Sarepta Therapeutics – Duchenne Gene Therapy Update (Webinar Recording)

Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet Villa, cardiologist at Cincinnati Children’s Hospital, and Dr. Craig…

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Watch: Duchenne Newborn Screening – Next Steps Following ACHDNC Dissolution (Webinar Recording)

Last week, PPMD and the Muscular Dystrophy Association provided insights into the current status of the Duchenne nomination to the federal Recommended Uniform Screening Panel (RUSP) for newborn screening following the dissolution of the Advisory…

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Watch: Wave Life Sciences – Update on FORWARD-53 Study of WVE-N531 (Webinar Recording)

Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the company’s Phase 2 FORWARD-53 trial of WVE-N531, Wave’s exon skipping oligonucleotide being investigated for the treatment of individuals with…

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Watch: Avidity Biosciences – Topline Data from EXPLORE44® Clinical Trial (Webinar Recording)

Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their EXPLORE44® clinical trial, which is assessing the safety and efficacy of the investigational therapy delpacibart zotadirsen (formerly AOC 1044, abbreviated…

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WATCH: Capricor Therapeutics – Update on Deramiocel for Cardiomyopathy in Patients with Duchenne (Webinar Recording)

Capricor Therapeutics recently joined PPMD for a community webinar to provide an update on their novel cell therapy, deramiocel (formerly CAP-1002), targeting cardiomyopathy in individuals living with Duchenne. The Capricor team discussed their ongoing Biologics…

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PPMD Hosts Duchenne Early Intervention Symposium

This May, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) plans to vote on whether to add Duchenne to the Recommended Uniform Screening Panel (RUSP) for newborn screening. To support a push…

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February 28, 2025

WATCH: Solid Biosciences – Update on Gene Therapy Candidate SGT-003 (Webinar Recording)

Members of the Solid Biosciences team recently joined PPMD for a community webinar to review initial positive data from the INSPIRE DUCHENNE study of SGT-003, a next-generation microdystrophin gene therapy candidate. The Solid team provided…

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December 19, 2024

WATCH: Sevasemten – CANYON Trial Topline Results (Webinar Recording)

Edgewise Therapeutics recently joined PPMD for a breaking news community webinar, during which Edgewise provided an update on the recently released topline results from its CANYON double-blind, randomized, placebo-controlled design to investigate the effect of…

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