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 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 for pregnant women and their babies. Researchers track serious side effects and disease complications. The goal is to better understa…
Matched conditions: LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New oil therapy could slash Life-Threatening events in kids with rare metabolic disease
Disease control OngoingThis study tests whether triheptanoin, a special oil, can reduce major clinical events (like severe low blood sugar or heart problems) in children with long-chain fatty acid oxidation disorders (LC-FAOD), a rare condition where the body can't break down certain fats for energy. A…
Matched conditions: LONG-CHAIN FATTY ACID OXIDATION DISORDERS (LC-FAOD)
Phase: PHASE3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC