Which medications are FDA-approved for the treatment for spinal muscle atrophy (SMA)?

Updated: Aug 11, 2020
  • Author: Ashish S Ranade, MBBS, MS, MRCS; Chief Editor: Jeffrey A Goldstein, MD  more...
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Answer

In December 2016, the US Food and Drug Administration (FDA) approved nusinersen, the first drug approved to treat children (including newborns) and adults with spinal muscular atrophy (SMA). In May 2019, the recombinant AAV9-based gene therapy onasemnogene abeparvovec was approved for type I SMA in children aged 2 years or younger. In August 2020, risdiplam, an SMN2 splicing modifier, was approved for type I, II, and III SMA in adults and children aged 2 months or older.


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