New hope for SMA patients: early access to apitegromab
Disease control
NO_LONGER_AVAILABLE
This program gives eligible patients with spinal muscular atrophy (SMA) early access to the investigational drug apitegromab before it is officially approved. Participants must be at least 2 years old and have a confirmed SMA diagnosis. A doctor will decide if the potential benef…
Sponsor: Scholar Rock, Inc. • Aim: Disease control
Last updated May 15, 2026 11:53 UTC