Child Neurology: Pathologically Confirmed Thrombotic Microangiopathy Caused by Onasemnogene Abeparvovec Treatment for SMA
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DOI: 10.1212/wnl.0000000000200676
Abstract: Onasemnogene abeparvovec is an adeno-associated virus vector-based gene therapy for spinal muscular atrophy (SMA). Although several cases of drug-induced thrombotic microangiopathy due to onasemnogene abeparvovec have been reported, none has been confirmed pathologically. Here, we… read more here.
Keywords: thrombotic microangiopathy; neurology; abeparvovec treatment; microscopy ... See more keywords