Early access to experimental SMA drug apitegromab offered before approval
Disease control
NO_LONGER_AVAILABLE
This expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed…
Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC