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). [electronic resource]
Producer: 20200401Description: 169-177 p. digitalISSN:- 1573-2665
- Adolescent
- Adult
- Cardiomyopathies -- metabolism
- Child
- Child, Preschool
- Fatty Acids -- metabolism
- Female
- Humans
- Hypoglycemia -- metabolism
- Infant
- Infant, Newborn
- Lipid Metabolism, Inborn Errors -- drug therapy
- Male
- Middle Aged
- Oxidation-Reduction -- drug effects
- Retrospective Studies
- Rhabdomyolysis -- metabolism
- Triglycerides -- administration & dosage
- Young Adult
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Publication Type: Clinical Trial, Phase II; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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