Pivotal SAPPHIRE Trial Data Published in The Lancet Neurology: Apitegromab Demonstrated Significant Motor Function Gains for Children and Adults with SMA on SMN-Targeted Treatment - Candlesense

Pivotal SAPPHIRE Trial Data Published in The Lancet Neurology: Apitegromab Demonstrated Significant Motor Function Gains for Children and Adults with SMA on SMN-Targeted Treatment

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other severe and debilitating neuromuscular diseases, announced today that positive study results from the pivotal Phase 3 SAPPHIRE trial (NCT05156320) were published in the peer-reviewed journal The Lancet Neurology. The data showed that children and adults with SMA had improve