Glucose Transporter Type 1 Deficiency Syndrome
0
Pipeline Programs
1
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
0
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Ultragenyx PharmaceuticalNOVATO, CA
2 programsTriheptanoinPHASE_21 trial
UX007PHASE_21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalUX007
Ultragenyx PharmaceuticalTriheptanoin
Clinical Trials (2)
Total enrollment: 18 patients across 2 trials
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
Start: Sep 2015Est. completion: Oct 201915 patients
Phase 2Terminated
Pilot Study of Triheptanoin in Patients With Glucose Transporter 1 Deficiency Syndrome
Start: Apr 2014Est. completion: Oct 20163 patients
Phase 2Unknown
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.