Onasemnogene Abeparvovec
CAS nullBiologic
Overview
Onasemnogene Abeparvovec is a gene therapy drug used to treat spinal muscular atrophy (SMA), a severe neuromuscular disease caused by a genetic mutation. It delivers a functional copy of the SMN1 gene, which helps in the production of the survival motor neuron protein.
Therapeutic areas
NeurologyGenetic Disorders
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