Bezafibrate for an inborn mitochondrial beta-oxidation defect

N Engl J Med. 2009 Feb 19;360(8):838-40. doi: 10.1056/NEJMc0806334.
No abstract available

Publication types

  • Evaluation Study
  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Bezafibrate / therapeutic use*
  • Carnitine O-Palmitoyltransferase / deficiency*
  • Carnitine O-Palmitoyltransferase / metabolism
  • Genes, Recessive
  • Humans
  • Hypolipidemic Agents / therapeutic use*
  • Lipid Metabolism, Inborn Errors / drug therapy*
  • Lipid Metabolism, Inborn Errors / metabolism
  • Mitochondrial Diseases / drug therapy*
  • Mitochondrial Diseases / metabolism
  • Oxidation-Reduction
  • Palmitoylcarnitine / metabolism
  • Pilot Projects
  • Quality of Life
  • RNA, Messenger / metabolism
  • Rhabdomyolysis / prevention & control
  • Statistics, Nonparametric

Substances

  • Hypolipidemic Agents
  • RNA, Messenger
  • Palmitoylcarnitine
  • Carnitine O-Palmitoyltransferase
  • Bezafibrate