614PCANYON trial results: sevasemten, an investigational fast skeletal myosin inhibitor, reduced muscle damage biomarkers and stabilized function in BMD

C. McDonald, J. Donovan(Healthwise), James E. MacDougall(BioBridges (United States)), E. Niks(Leiden University Medical Center), Michela Guglieri(Muscular Dystrophy UK), D. Castro(University of North Texas), H. Kushlaf(University of Cincinnati Medical Center), Banny S. Wong(UMass Memorial Health Care), J. Statland(University of Kansas Medical Center), K. Mathews(University of Iowa), Vidhyapriya Sreenivasan(University of Colorado Denver), Roxana Donisa Dreghici(Roche (Switzerland)), Nicholas Johnson(Animal and Plant Health Agency), Doris G. Leung(Kennedy Krieger Institute), Rosaline C. M. Quinlivan(National Hospital for Neurology and Neurosurgery), A. Veerapandiyan(Arkansas Children's Hospital), Alan J. Russell(Healthwise), H. Phan(Rare Disease Therapeutics (United States)), A. Connolly(Nationwide Children's Hospital), Atul Varadhachary(Washington University in St. Louis)
Neuromuscular Disorders
September 1, 2025
Cited by 0


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