LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)
Clinical trials for LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD) explained in plain language.
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New drug aims to cut life-threatening events in kids with rare metabolic disease
Disease control OngoingThis study tests whether triheptanoin, a special fat-based medicine, works better than standard MCT oil at preventing major health crises in children with long-chain fatty acid oxidation disorders (LC-FAOD). These rare genetic conditions prevent the body from breaking down certai…
Matched conditions: LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 22, 2026 14:03 UTC
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New study tracks Long-Term safety of rare disease treatment
Disease control OngoingThis study follows 150 people with long-chain fatty acid oxidation disorders (LC-FAOD) to check the long-term safety of their treatment, including during pregnancy and in infants. Researchers will track serious side effects and disease complications. The goal is to better underst…
Matched conditions: LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 20, 2026 11:54 UTC