DRUGinvestigationalsmall molecule
triheptanoin
Brand names: Dojolvi
Mechanism
Anaplerotic substrate for energy metabolism. Positive in SMND7 mice (2021) — both survival and motor.. NOTE: Approved 2020 as Dojolvi (Ultragenyx) for LC-FAOD (long-chain fatty acid oxidation disorders); SMA use is off-label/investigational.
Approved indications
LC-FAOD (off-label for SMA)
Related claims (0)
No claims matched “triheptanoin” in free-text search.
Off-Target Findings (0)
No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.