Long-chain Fatty Acid Oxidation Disorders
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS other · 2026-04-13Clinical Trial Update: Expanded Access to Triheptanoin
Expanded access to triheptanoin is available for patients with long-chain fatty acid oxidation disorders, according to the clinical trials r…
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CLINICAL_TRIALS research · 2026-04-07Clinical Trial Update: A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD [Phase 3]
A Phase 3 clinical trial compares triheptanoin with even-chain medium-chain triglycerides to assess metabolic crisis events in pediatric pat…
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CLINICAL_TRIALS trial_result · 2026-03-06Clinical Trial Update: Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
The update indicates Ultragenyx's in-clinic disease monitoring program for long-chain fatty acid oxidation disorders is currently active but…
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CLINICAL_TRIALS research · 2024-08-23Clinical Trial Update: Exercise Intolerance Study in Long Chain Fatty Acid Oxidation Disorders
A clinical trial update announcing a recruiting study evaluating exercise intolerance in individuals with Long-chain Fatty Acid Oxidation Di…
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FDA approval · 2023-10-30FDA Approved: DOJOLVI — Supplemental Application (Ultragenyx Pharmaceutical Inc.)
The FDA approved a supplemental application for DOJOLVI (triheptanoin) submitted by Ultragenyx Pharmaceutical Inc. The action pertains to it…
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CLINICAL_TRIALS trial_result · 2021-02-11Clinical Trial Update: A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) [Phase 2]
Phase 2 clinical trial evaluating UX007 (triheptanoin) in participants with long-chain fatty acid oxidation disorders (LC-FAOD) has complete…
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FDA approval · 2020-06-30FDA Approved: DOJOLVI — Original Application (Ultragenyx Pharmaceutical Inc.)
The FDA approved Dojolvi (triheptanoin) from Ultragenyx Pharmaceutical Inc. for the treatment of long-chain fatty acid oxidation disorders i…
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CLINICAL_TRIALS withdrawal · 2014-07-28Clinical Trial Update: Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects [Phase 3]
A Phase 3 trial of Triheptanoin for long-chain fatty acid oxidation disorders has been withdrawn.