Ultragenyx gains on De Vivo results
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) gained $2.74 Wednesday to $66.96 after announcing positive results from an investigator-sponsored Phase II study in six evaluable patients with glucose transporter type-1 deficiency syndrome (Glut1 DS; De Vivo disease). Ultragenyx's triheptanoin ( UX007) reduced the incidence of paroxysmal manifestations by 90% (p=0.028). Paroxysmal manifestations rebounded significantly (p=0.043) during a two-month withdrawal phase after treatment.
Deficiency of solute carrier family 2 facilitated glucose transporter member 1 ( SLC2A1; GLUT1) limits glucose delivery to the brain, causing seizures and movement disorders. Triheptanoin is a synthetic compound that can be metabolized to replace intermediate substrates in fatty acid oxidation and the tricarboxylic acid (TCA) cycle. ...