Last Updated: 09/26/2026
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Found 4 publications
Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies.
Journal: Blood advances
Published: October 29, 2024
Dietary management and major clinical events in patients with long-chain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study.
Journal: Clinical nutrition ESPEN
Published: November 09, 2020
Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study.
Journal: Journal of inherited metabolic disease
Published: January 27, 2020
Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD).
Journal: Journal of inherited metabolic disease
Published: February 12, 2019
Showing 1-4 of 4
Last Updated: 09/26/2026