Ultragenyx Announces First Patient Enrolled in Phase 2 Study of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome

News | 03. 11. 2014

Ultragenyx Pharmaceutical

Novato, CA, March 11, 2014 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient enrolled in the Phase 2 study of triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS), at Columbia University. Glut1 DS, also known as De Vivo disease, is a rare, severely debilitating disease characterized by seizures, developmental delay, and movement disorder.