Long-term follow-up in an open-label trial of triheptanoin in GLUT1 deficiency syndrome: a sustained dramatic effect

J Neurol Neurosurg Psychiatry. 2019 Nov;90(11):1291-1293. doi: 10.1136/jnnp-2018-320283. Epub 2019 Apr 4.
No abstract available

Keywords: GLUT1 deficiency syndrome; movement disorders; triheptanoin.

Publication types

  • Clinical Trial
  • Letter
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Carbohydrate Metabolism, Inborn Errors / drug therapy*
  • Follow-Up Studies
  • Humans
  • Monosaccharide Transport Proteins / deficiency*
  • Triglycerides / therapeutic use*

Substances

  • Monosaccharide Transport Proteins
  • Triglycerides
  • triheptanoin

Supplementary concepts

  • Glut1 Deficiency Syndrome