Triheptanoin for the treatment of glucose transport 1 deficiency

摘要

Provided are methods for treating GLUT1 and related brain energy deficiencies comprising administering odd-carbon fatty acid sources, e.g., C5 or C7 fatty acid sources, and related compositions.

主权项

1. A method of treating a movement disorder associated with GLUT1 deficiency in a human subject in need thereof, comprising administering to the subject an odd-chain fatty acid source, wherein the movement disorder is selected from ataxia, chorea, spasticity, dystonia, and paroxysmal exercise-induced dyskinesia.