Spinal Muscle Atrophy Medication

Updated: Nov 05, 2018
  • Author: Ashish S Ranade, MBBS, MS, MRCS; Chief Editor: Jeffrey A Goldstein, MD  more...
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Medication

Medication Summary

In December 2016, the US FDA approved nusinersen, the first drug approved to treat children (including newborns) and adults with spinal muscular atrophy (SMA).

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Antisense Oligonucleotides

Class Summary

Antisense oligonucleotides (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency may be considered for treatment.

Nusinersen (Spinraza)

In studies in transgenic animal models of SMA, nusinersen was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein. It is indicated for SMA in pediatric and adults patients.

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