Risdiplam in non-sitter patients aged 16 years and older with 5q spinal muscular atrophy.

This series suggests that risdiplam is safe and may be effective in non-sitter SMA patients older than 16 years. In these patients, functional scales and GAS would be more sensitive than motor scales to detect changes, because they include axial, bulbar and respiratory domains. Larger studies are needed to confirm these results.