More is needed to complement the available therapies of spinal muscular atrophy

Future Med Chem. 2019 Nov;11(22):2873-2876. doi: 10.4155/fmc-2019-0239. Epub 2019 Oct 31.
No abstract available

Keywords: AAV9; ISS-N1; Spinraza; gene therapy; nusinersen; oligonucleotide; spinal muscular atrophy (SMA); survival motor neuron (SMN).

Publication types

  • Editorial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Exodeoxyribonucleases / genetics
  • Genetic Therapy
  • Humans
  • Muscular Atrophy, Spinal / genetics
  • Muscular Atrophy, Spinal / therapy*

Substances

  • DCLRE1B protein, human
  • Exodeoxyribonucleases