Subacute liver injury in two young infants following gene replacement therapy for spinal muscular atrophy

Abstract excerpt

Spinal muscular atrophy is a neurodegenerative disorder resulting from the irreversible loss of anterior horn cells secondary to homozygous mutations in the survival motor neuron gene SMN1. Gene replacement therapy using a recombinant adeno-associated virus 9 vector containing an SMN1 gene construct, onasemnogene abeparvovec-xioi, was approved by the US Food and Drug Administration in May 2019. Subacute mild...

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