Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy following onasemnogene abeparvovec. Early recognition and treatment of drug-induced thrombotic microangiopathy may lessen mortality and morbidity.

Automated summary

Spinal muscular atrophy is treated with onasemnogene abeparvovec, but it can lead to drug-induced thrombotic microangiopathy in some infants. Early recognition and treatment of this complication may reduce mortality and morbidity.