Emmanuel Roze

Oct 19, 2024 | movementdisorders.onlinelibrary.wiley.com | Emmanuel Roze |Institut du Cerveau |AP-HP Paris France

The Ultragenyx@-sponsored phase 3 randomized controlled study evaluating the effect of triheptanoin to treat paroxysmal episodes of movement disorders related to Glut1 deficiency syndrome (Glut1-DS) failed to demonstrate efficacy.1 This was unexpected considering the rationale supporting the use of triheptanoin,2 and the striking results of preliminary investigator-driven open-label studies that showed over 90% reduction of paroxysmal movement disorders in patients with Glut1-DS,3, 4 a...