Triheptanoin for the treatment of glucose transport 1 deficiency
AssigneesNational Institute of Health and Medical Research, Baylor Research Institute
InventorsFanny MOCHEL, Raphael SCHIFFMANN
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Abstract
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.
Claims38
A method of treating a GLUT1 deficiency or a GLUT1 deficiency-associated condition or disorder in a human subject in need thereof, comprising administering to the subject an odd-chain fatty acid source.
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A method of treating or preventing diseases of the brain affecting energy metabolism in a subject, comprising administering to the subject an effective amount of triheptanoin, where the administration of triheptanoin restores mitochondrial energy function and net biosynthesis through anaplerosis.
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Dependencies are inferred from the claim text.