Conclusion
risdiplam, Risdiplam, later-onset SMA, upper limb motor function
DISEASE
Risdiplam treatment in a broad and clinically heterogeneous patient population of children, teenagers, and adults with later-onset SMA and varying disease duration resulted in continued clinically relevant gains in motor function. This was demonstrated by improvements (32% of patients) and stabilization (58% of patients) in MFM32 total score at month 24 and confirmed by progressive improvements on RULM—an additional, independent measure of upper limb motor function—as well as caregiver-reported SMAIS‑ULM. The gains in motor function observed after 12 months of risdiplam treatment were maintained or improved upon after 24 months, confirming the benefit of longer-term treatment with risdiplam. The safety profile after 24 months of treatment was consistent with that previously observed after 12 months of treatment [