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  • Updated 03.21.2021
  • Released 03.08.2018
  • Expires For CME 03.21.2024



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.

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