Nusinersen therapy has demonstrated clinically meaningful benefits with improved motor skills, respiratory function, survival, and other outcomes in infants, children, and adults with spinal muscular atrophy (SMA).

We report interim safety, efficacy, and pharmacokinetics results from PANDA (NCT04419233), a phase 4 post-marketing surveillance study of people with SMA treated with nusinersen in routine clinical practice in China.

Fifty participants were enrolled in PANDA as of January 4, 2023, all receiving at least one dose of nusinersen.

Adverse events (AEs, primary endpoint) were experienced by 45 (90%) participants and were mostly mild/moderate; no AEs led to nusinersen discontinuation or study withdrawal.

Nusinersen was generally well tolerated with an acceptable overall safety profile, consistent with the known safety of nusinersen; efficacy, safety, and nusinersen exposure are consistent with prior observations.