Clinical trial reveals encouraging results for apitegromab in spinal muscular atrophy
Mihaela Măncilă
Ann & Robert H. Lurie Children's Hospital of Chicago Sep 18 2025 A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study published in The Lancet Neurology. Spinal muscular atrophy (SMA) is a rare genetic disorder that affects motor neurons - nerve cells in the spinal cord responsible for controlling voluntary muscle movement. Caused by mutations in the
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