Combination therapy with nusinersen and AVXS-101 in SMA type 1

Neurology. 2019 Oct 1;93(14):640-641. doi: 10.1212/WNL.0000000000008207. Epub 2019 Sep 5.
No abstract available

Publication types

  • Case Reports
  • Observational Study

MeSH terms

  • Combined Modality Therapy / methods
  • Genetic Therapy / methods*
  • Humans
  • Infant
  • Male
  • Oligonucleotides / administration & dosage*
  • Spinal Muscular Atrophies of Childhood / diagnosis
  • Spinal Muscular Atrophies of Childhood / genetics*
  • Spinal Muscular Atrophies of Childhood / therapy*

Substances

  • Oligonucleotides
  • nusinersen