UX007 (highly purified synthetic 7-carbon fatty acid triglyceride):
Phase 3 movement disorder study
Ultragenyx Pharmaceutical Inc. is conducting a global Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 (highly purified synthetic 7-carbon fatty acid triglyceride) in the treatment of paroxysmal movement disorders associated with Glut1 DS.
The study will enroll individuals diagnosed with Glut1 DS who are at least 6 years old and who experience movement disorder events which impact or limit physical functioning and/or activities of daily living such as caring for oneself, walking, speaking, sleeping, and participating in school/work or doing sports/activities.
The study will evaluate safety and efficacy including changes in the frequency of movement disorder events, walking capacity, cognition, and other quality of life measures.
For more information on how to get involved, visit:
This treatment is investigational and has not yet been approved for use by any regulatory agency. Efficacy and safety have not been established. The information presented should not be construed as a recommendation for use.
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