Patient ≥2 yr weighing ≥20 kg 5 mg once daily,<20 kg 0.25 mg/kg once daily;2 mth to <2 yr 0.20 mg/kg once daily; <2 mth 0.15 mg/kg once daily. Max: 5 mg daily.
Patients who previously received SMN1 gene therapy. Retinal toxicity. Not to be taken by patients w/ rare hereditary problems of fructose intolerance. Patients of reproductive potential should use highly effective contraception during treatment & until at least 1 mth after last dose in female patients & 4 mth after last dose in male patients w/ female partners of childbearing potential. Male patients should not donate sperm during treatment & for 4 mth after last dose. Not recommended during pregnancy & lactation. Increased jaundice in newborn infants up to 4 wk old.