Ultragenyx Initiates New Development Program Studying Triheptanoin (UX007) for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

News | 08. 05. 2013

Ultragenyx Pharmaceutical

Phase 2 Trial Initiation Planned for Year End

NOVATO, Calif., Aug 05, 2013 (GLOBE NEWSWIRE via COMTEX) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced its plan to develop triheptanoin (UX007) for the treatment of seizures in glucose transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS is a rare and severely debilitating disease caused by mutations in the SLC2A1 gene, which encodes for a protein called glucose transporter type-1 (Glut1). The disease is characterized by seizures, developmental delay and movement disorders. The Glut1 protein transports glucose from blood into the brain. Because glucose is the primary source of energy for the brain, this defect in the Glut1 transporter results in a chronic state of brain energy deficiency. The company is planning to initiate a Phase 2 trial studying triheptanoin in Glut1 DS by the end of 2013.