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1.
Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study.
J Inherit Metab Dis
; 44(1): 253-263, 2021 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-32885845
2.
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).
J Inherit Metab Dis
; 42(1): 169-177, 2019 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-30740733
3.
Dietary management and major clinical events in patients with long-chain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study.
Clin Nutr ESPEN
; 41: 293-298, 2021 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-33487279
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