Long-term follow-up in an open-label trial of triheptanoin in GLUT1 deficiency syndrome: a sustained dramatic effect
Author:
Funder
Ultragenyx Pharmaceutical Inc.
Agence Nationale de la Recherche
Publisher
BMJ
Subject
Psychiatry and Mental health,Neurology (clinical),Surgery
Reference10 articles.
1. Defective Glucose Transport across the Blood-Brain Barrier as a Cause of Persistent Hypoglycorrhachia, Seizures, and Developmental Delay
2. Glucose transporter-1 deficiency syndrome: the expanding clinical and genetic spectrum of a treatable disorder
3. Long-Term Clinical Course of Glut1 Deficiency Syndrome
4. The spectrum of movement disorders in Glut-1 deficiency
5. Glut1 Deficiency Syndrome and Novel Ketogenic Diets
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2. 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;Movement Disorders;2024-05-09
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