Ultragenyx Investigational New Drug Application for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome is in Effect
Phase 2 Clinical Study Expected to Begin in the First Half of 2014
NOVATO, CA – December 17, 2013 – Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced today that its Investigational New Drug (IND) application
filing for triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS) is in effect. The IND allows the company to proceed with its clinical development program for triheptanoin in Glut1 DS in the United States. The company plans to initiate a Phase 2 trial studying triheptanoin in Glut1 DS in the first half of 2014.
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