UX007
PRECLINICAL
PHASE 1
PHASE 2
PHASE 3
PRE
P1
P2
P3
Pipeline Type
Dietary Supplements
Phase Of Development
Phase 2
Sponsors and Collaborators
Ultragenyx
Mechanism of Action
Mechanism Description
highly purified synthetic 7-carbon fatty acid triglyceride
UX007 metabolized into heptanoate; can cross blood-brain barrier to provide energy to the brain. Heptanoate and five-carbon ketone bodies may also regenerate new glucose in the brain
Populations Tested In
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Other Resources
Special FDA Designation
Nonprofit or Government Support
N/A
Nonprofit or Government Support Description
N/A
Previous Presentations
Presented at meetings (latest date) (Pipeline, AEDD; AES, Eilat, etc)
N/A
Location of Clinical Trials
US: 5 sites, UK, France, Israel,
Australia: Victoria, Italy, Spain
Clinical Trial link