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 ...

After all the anticipation, Novartis has the FDA approval it has been seeking for Zolgensma, its gene therapy for spinal muscular atrophy, and has priced it at the low end of expectations. The FDA ...

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.

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

And on the flip side, why can adults with spinal muscular atrophy (SMA) access disease-modifying therapies in Quebec, while in most other provinces they remain excluded entirely? These inconsistencies ...