Triheptanoin (UX-007) for glucose transporter type 1 deficiency syndrome (de vivo disease) – first line


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Year: 2017

Glucose transporter type 1 deficiency syndrome (Glut1 DS) is a rare genetic disorder that affects how the body moves glucose (used for energy) into the brain. The most common symptom of this condition is seizures (epilepsy), which usually begin within the first few months of life. However, the symptoms and severity of Glut1 deficiency syndrome can vary substantially from one person to another.
Triheptanoin is a novel drug being developed to reduce seizures and other symptoms in patients with Glut1 DS. The drug acts by producing a substitute compound that can produce glucose in the brain. The safety and efficacy of triheptanion is currently being evaluated. If marketed this will become the first licensed treatment in reduction of seizures in patients with Glut1 DS.