Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx)1, an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1,

Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today 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