Advanced therapies in rare diseases: the example of spinal muscular atrophy

Med Clin (Barc). 2018 Oct 12;151(7):275-277. doi: 10.1016/j.medcli.2018.03.001. Epub 2018 Apr 21.
[Article in English, Spanish]
No abstract available

Publication types

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

MeSH terms

  • Child
  • Child, Preschool
  • Humans
  • Infant
  • Muscular Atrophy, Spinal / drug therapy*
  • Rare Diseases / drug therapy*