New hope for rare metabolic disorder patients through expanded drug access
NCT ID NCT03773770
First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 21 times
Summary
This program provides expanded access to triheptanoin for people with long-chain fatty acid oxidation disorders who have few treatment choices and cannot join a clinical trial. It aims to help manage the disease by supplying an alternative energy source. Participants must not be eligible for any ongoing UX007 clinical trial.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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