Onasemnogene abeparvovec

K K Jain MD (Dr. Jain is a consultant in neurology and has no relevant financial relationships to disclose.)
Originally released May 25, 2020; expires May 25, 2023

This article includes discussion of onasemnogene abeparvovec, AVXS-101, onasemnogene, onasemnogene abeparvovec, onasemnogene abeparvovec-xioi, and Zolgensma. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.

Historical note and terminology

Onasemnogene abeparvovec is a gene therapy for delivery of a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy type 1 and is the first gene therapy to be approved for the treatment of spinal muscular atrophy in the United States in May 2019 (Hoy 2019). It was approved by the European Commission in May 2020. It will compete with intrathecal nusinersen, a splice-modulating antisense oligonucleotide, approved for the treatment of spinal muscular atrophy type 1 in 2016.

The content you are trying to view is available only to logged in, current MedLink Neurology subscribers.

If you are a subscriber, please log in.

If you are a former subscriber or have registered before, please log in first and then click select a Service Plan or contact Subscriber Services. Site license users, click the Site License Acces link on the Homepage at an authorized computer.

If you have never registered before, click Learn More about MedLink Neurology  or view available Service Plans.

Find out how you can join MedLink Neurology