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Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
Deepa H Chand1, Craig Zaidman2, Kapil Arya3
1Novartis Gene Therapies, Bannockburn, IL; Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO.
Abstract:
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.
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