May 2024

The following content is sponsored by Edgewise Therapeutics and does not reflect the views of Muscular Dystrophy News or BioNews, Inc. Becker muscular dystrophy (Becker) is a serious genetic disease that imposes significant physical, emotional, financial, and social challenges on affected individuals and their families. Symptoms of Becker, including muscle wasting and cardiopulmonary deficits, can…

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Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO to enable skipping of exon 51 with a fragment antibody (Fab) to increase targeted delivery of the product to skeletal…

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PPMD is pleased to share that the article, “Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals,” has been published in the Orphanet Journal of Rare Diseases. This qualitative study aims to understand the barriers faced by Hispanic/Latino families specifically and underrepresented groups more generally to clinical trial…

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DYNE-101 and DYNE-251, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, continue to work as intended and may lead to functional improvements for patients, according to new clinical trial data. These findings come from ongoing Phase 1/2 studies, which may still be recruiting. DELIVER (NCT05524883), launched in 2022, is testing DYNE-251 in boys ages…

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As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates in their next-generation exon skipping program for Duchenne amenable to skipping exon 51.  In their phase 1/2 Trial, Dyne-251 at dose of 10 mg/kg given once a month showed an average 3.2% normal dystrophin expression (7.6% normal dystrophin when adjusted…

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PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren), and has returned the opinion to the CHMP for re-evaluation.  As a result, Translarna, a…

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Regular readers of my column will know that my wife, Wendy, and I moved from Shanksville, Pennsylvania, to Pittsburgh in January 2023 to be closer to our children and grandchildren. There have been some issues, but the move has worked out well. We see a lot more of our kids, for instance. But I have…

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**UPDATE**  On May 10, 2024, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) decided to postpone the vote on including Duchenne Muscular Dystrophy (DMD) in the Recommended Uniform Screening Panel (RUSP). The committee seeks additional information to ensure an evidence-based decision. This decision will be revisited no later than one year from…

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Findings from a Phase 2 trial testing losmapimod in adults with facioscapulohumeral muscular dystrophy (FSHD) have offered new insights into the best outcome measures for future clinical trials, according to final published data from the clinical study, which wrapped up in 2021. The  Fulcrum Therapeutics treatment, as previously reported, did not significantly reduce signs of…

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This week marked a significant milestone as Congress successfully passed the bipartisan Federal Aviation Administration (FAA) reauthorization, officially known as HR 3935, the Securing Growth and Robust Leadership in American Aviation Act. This legislation, now making its way to President Biden’s desk to be signed into law, represents a groundbreaking advancement in air travel accessibility,…

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