K K Jain MD (Dr. Jain is a consultant in neurology and has no relevant financial relationships to disclose.)
Originally released March 8, 2018; last updated March 9, 2020; expires March 9, 2023

This article includes discussion of BIIB058, Ionis-SMNRx, ISIS 396443, and ISIS-SMNRx. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.

Historical note and terminology

Nusinersen is a splice-modulating antisense oligonucleotide indicated for the treatment of spinal muscular atrophy. Intrathecal nusinersen was approved by the U.S. Food and Drug Administration (FDA) in 2016. Another splice-modulating oligonucleotide, eteplirsen, was also approved earlier in 2016 for the treatment of Duchenne muscular dystrophy. The FDA granted fast track designation and priority review to the application for nusinersen. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Pharmacological data and results of clinical trials submitted for the approval of nusinersen can be viewed on the FDA website. Nusinersen was approved as a treatment for spinal muscular atrophy in the European Union in 2017.

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