Scholar Rock publishes data on inhibiting myostatin activation in models of SMA
Scholar Rock announced the publication, "Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy" in the peer-reviewed journal Human Molecular Genetics. The publication details preclinical studies demonstrating that a highly specific inhibitor of myostatin activation effectively increased muscle mass and function in mouse models of Spinal Muscular Atrophy, or SMA. In addition, despite baseline serum levels of inactive latent myostatin being lower in the SMA mice than in mice without disease, successful target engagement was observed as evidenced by increased levels of latent myostatin in serum following treatment. Together, the results support the potential for myostatin blockade as a therapeutic strategy to address motor functional deficits in SMA.