Advances in the Therapy of Spinal Muscular Atrophy

J Pediatr. 2021 Sep:236:13-20.e1. doi: 10.1016/j.jpeds.2021.06.033. Epub 2021 Jun 29.
No abstract available

Keywords: nusinersen; onasemnogene abeparvovec; risdiplam.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adolescent
  • Adult
  • Azo Compounds / therapeutic use
  • Biological Products / therapeutic use
  • Child
  • Child, Preschool
  • Humans
  • Infant
  • Infant, Newborn
  • Neuromuscular Agents / therapeutic use
  • Oligonucleotides / therapeutic use
  • Pyrimidines / therapeutic use
  • Recombinant Fusion Proteins / therapeutic use
  • Spinal Muscular Atrophies of Childhood / diagnosis
  • Spinal Muscular Atrophies of Childhood / etiology
  • Spinal Muscular Atrophies of Childhood / therapy*
  • Young Adult

Substances

  • Azo Compounds
  • Biological Products
  • Neuromuscular Agents
  • Oligonucleotides
  • Pyrimidines
  • Recombinant Fusion Proteins
  • Zolgensma
  • nusinersen
  • Risdiplam