Triheptanoin Did Not Show Benefit versus Placebo for the Treatment of Paroxysmal Movement Disorders in Glut1 Deficiency Syndrome: Results of a Randomized Phase 3 Study.
Valentina De GiorgisKailash P BhatiaOdile Boespflug-TanguyDomitille GrasAdela Della MarinaArchana DesurkarManuel ToledoIan MillerMichael RotsteinSusanne A SchneiderDaniel C TarquinioYvonne WeberMelanie BrandaburJill MayhewTony KoutsoukosDarryl C De VivoPublished in: Movement disorders : official journal of the Movement Disorder Society (2024)
There were no significant differences between the triheptanoin and placebo groups in the frequency of disabling movement disorder events during the double-blind maintenance period. © 2024 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.