Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency
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Pipeline Programs
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Companies
1
Clinical Trials
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Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalUX007
Clinical Trials (1)
Total enrollment: 94 patients across 1 trials
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
Start: Dec 2014Est. completion: Dec 202094 patients
Phase 2Completed
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.