Abstract
Onasmnogene abeparvovec (OA) is a recently approved gene therapy for spinal muscular atrophy (SMA),1 administered as a single intravenous infusion. Liver toxicity is the main safety concern.2 We report on a patient with type 1 SMA, previously treated with nusinersen, with a transient marked increase in creatine kinase (CK) levels, consistent with acute myositis, after switching to OA.
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