Scholar Rock, Inc.

This study is testing an investigational drug called apitegromab in infants under 2 years old who have spinal muscular atrophy (SMA). The main goal is to see how the drug moves through the body, how it affects a specific muscle protein, and if it can improve gross motor skills, l…

This is the first study in humans for a new drug called SRK-439. It aims to check if the drug is safe and how it behaves in the body when given to healthy adults. Researchers will give different doses to small groups of volunteers to see how well it is tolerated and measure its e…