[HTML][HTML] Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series
DH Chand, C Zaidman, K Arya, R Millner… - The Journal of …, 2021 - Elsevier
The Journal of pediatrics, 2021•Elsevier
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.
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.
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.
Elsevier