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Australian company Percheron Therapeutics released topline 6-month data from its Phase 2 study of avicursen in non-ambulatory individuals with Duchenne. The trial did not meet its primary endpoint, and there were no clear trends toward benefits on secondary outcomes. Therefore, Percheron is discontinuing the trial, and will share more analyses next year.    READ MORE IN THE…

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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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The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases. Altogether, the funding totals more than $5 million across 21 grants, according to a press release from the nonprofit. “The Muscular Dystrophy Association is…

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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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