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CONy 2021 | Approved gene-based therapies for neuromuscular diseases

Aida Abu-Baker, PhD, Montreal Neurological Institute and Hospital, McGill University, Montreal, Canada, discusses gene therapy advances in neuromuscular diseases. Various gene-based therapies have been approved by the U.S. Food and Drug Administration (FDA) for spinal muscular atrophy (SMA), including nusinersen, onasemnogene abeparvovec, and more recently risdiplam. Significant advances have also been made in the treatment of Duchenne muscular dystrophy with the development and approval of eteplirsen. This interview was conducted during the virtual 2021 World Congress on Controversies in Neurology (CONy).