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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. 2019 Jan;42(1):169-177. doi: 10.1002/jimd.12038.
J Inherit Metab Dis. 2019.
PMID: 30740733
Free PMC article.
Clinical Trial.
UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24weeks of treatment.
Vockley J, Burton B, Berry GT, Longo N, Phillips J, Sanchez-Valle A, Tanpaiboon P, Grunewald S, Murphy E, Humphrey R, Mayhew J, Bowden A, Zhang L, Cataldo J, Marsden DL, Kakkis E.
Vockley J, et al.
Mol Genet Metab. 2017 Apr;120(4):370-377. doi: 10.1016/j.ymgme.2017.02.005. Epub 2017 Feb 7.
Mol Genet Metab. 2017.
PMID: 28189603
Free article.
Clinical Trial.
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