Learn what you need to do to have a fun, safe, and stress-free day at an amusement park when you have spinal muscular atrophy.

Objective The aim of this study was to assess early language acquisitions in treated individuals with spinal muscular atrophy (SMA) type 1 and in infants identified by newborn screening (NBS). Methods ...

Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, ...

Panelists discuss how intrathecal delivery of onasemnogene abeparvovec in the STEER study demonstrates statistically ...

Panelists discuss how gaps in SMA care persist despite highly effective treatments, particularly regarding racial and ethnic disparities in research participation and global access challenges due to ...