Zolgensma

Risk of acute serious liver injury and acute liver failure (esp. preexisting liver impairment or acute hepatic viral infection). ALT, AST, or total bilirubin levels (except due to neonatal jaundice) >2×ULN: not studied. Monitor liver function prior to infusion, weekly for the 1^st month, then every other week for the 2^nd/3^rd months until unremarkable results. Monitor creatinine, CBC prior to infusion. Monitor platelets and troponin-I prior to infusion, weekly for the 1^st month, then every other week (platelets) and monthly (troponin-I) for the 2^nd/3^rd months until levels return to baseline. Monitor for thrombotic microangiopathy (TMA); evaluate for hemolytic anemia and renal dysfunction if thrombocytopenia develops. Increased risk of serious systemic immune response; delay infusion in those with concurrent infections until resolved. Perform baseline anti-AAV9 antibody testing prior to infusion; may retest if titers are >1:50. Consult a specialist if acute serious liver injury/liver failure is suspected, troponin elevations accompanied by signs/symptoms, or TMA occurs. Premature neonates: delay infusion until full-term gestational age is reached.