December 2024

Greetings from Indiana! I write today letting you know that thanks to a generous pledge from a local family I know well, a $100,000 matching gift will be made to PPMD. That means every dollar donated between now and the end of the year will go twice as far. I hope I can convince you…

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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 sevasemten on individuals with Becker muscular dystrophy. Moderated by PPMD’s Pat Furlong, Edgewise’s VP, Patient Advocacy and External Innovation, Abby…

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As a pediatric neurologist who takes care of children with neuromuscular diseases, I’ve had the privilege of witnessing first-hand the benefit many of our therapies can have for individuals living with Duchenne. Your belief in PPMD and unwavering support has led to monumental progress for this community, and I am optimistic about what the future…

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Earlier this month, PPMD convened experts from across the country in Columbus, Ohio for a two-day workshop focused on the development of care standards for gastrointestinal, urological, and swallowing issues in Duchenne and Becker.  As part of an overarching effort to update the 2018 care considerations, PPMD’s Care Team was delighted to welcome a multidisciplinary…

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PPMD is disappointed to learn of Percheron Therapeutics’ recent decision to terminate the company’s ongoing Phase 2b randomized, placebo-controlled trial of avicursen (ATL1102) in non-ambulatory individuals living with Duchenne following the release of topline six-month results. Avicursen is an antisense oligonucleotide designed to reduce inflammation through inhibition of CD49d. Percheron reported that the trial did…

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Living with Duchenne muscular dystrophy (DMD) has involved a series of challenges and triumphs, each shaping the person I am today. As 2025 approaches, I’m reflecting on that journey as well as imagining the path ahead — especially since the coming year includes a significant milestone for me. I’ll turn 30 next year! That’s more…

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PPMD is disappointed to learn that PepGen Inc. has received a full clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical trial in patients with Duchenne in the United States. PGN-EDO51 is an PMO-exon skipping therapy bound to a peptide which…

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PepGen has received a clinical hold notice from the FDA regarding its Investigational New Drug (IND) application in Duchenne amenable to skipping exon 51, and will be unable to initiate their Phase 2 CONNECT2-EDO51 trial in the U.S, until that is resolved.  PepGen is currently conducting an open-label CONNECT1-EDO51 multiple ascending dose study in Canada,…

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Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in muscular dystrophies. According to Edgewise, the trial met its primary endpoint of change from…

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As an early funder of Edgewise, CureDuchenne is pleased to share that the Phase 2 Trial of sevasemten in Becker muscular dystrophy met its primary endpoint of reduction in creatine kinase (CK), a biomarker associated with skeletal muscle damage.  Individuals treated with sevasemten also showed stabilization of NSAA, with a trend towards improvement at 12…

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