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Welsh et al. featured in International Journal of Toxicology



An article by ToxStrategies’ scientist Dr. Brian Welsh and a team of colleagues at Scholar Rock, Inc., is featured in Volume 40 of the International Journal of Toxicology, with a figure from the article appearing on the cover. The team’s work consisted of a preclinical safety assessment of apitegromab, a novel therapy for the treatment of spinal muscular atrophy (SMA) in adults and children. The compound targets the myostatin pathway, a negative regulator of skeletal muscle that is a therapeutic target for patients with SMA. In vitro studies confirmed the ability of apitegromab to inhibit the activation of promyostatin. Toxicology studies in monkeys and in juvenile and adult rats for up to 26 weeks showed that weekly intravenous administration of apitegromab achieved sustained serum exposure and target engagement and was well-tolerated. The authors conclude that clinical studies of apitegromab in SMA patients are warranted