Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients

J Neurol Sci. 2016 Jul 15:366:65-67. doi: 10.1016/j.jns.2016.04.048. Epub 2016 Apr 26.
No abstract available

Keywords: 2-Hydroxypropyl-β-cyclodextrin; Intrathecal; Metabolic disease; Niemann-Pick; Toxicity.

Publication types

  • Case Reports
  • Letter

MeSH terms

  • 2-Hydroxypropyl-beta-cyclodextrin
  • Adult
  • Compassionate Use Trials
  • Fatal Outcome
  • Female
  • Humans
  • Injections, Spinal
  • Middle Aged
  • Niemann-Pick Disease, Type C / drug therapy*
  • Niemann-Pick Disease, Type C / genetics
  • beta-Cyclodextrins / administration & dosage*
  • beta-Cyclodextrins / adverse effects

Substances

  • beta-Cyclodextrins
  • 2-Hydroxypropyl-beta-cyclodextrin