SMA Newsroom

Scholar Rock announced on 11 September 2026 that the U.S. Food and Drug Administration (FDA) has approved apitegromab (Isembyld) for the treatment of SMA in adults and children aged 2 years and older who are receiving an SMN2-targeted treatment.

The European Commission has granted marketing authorisation for Itvisma (intrathecal formulation of onasemnogene abeparvovec, developed by Novartis) for the treatment of adults and children aged 2 years and older living with 5q spinal muscular atrophy (SMA).

We are pleased to launch SMA Europe’s Annual report 2025.