Effects of triheptanoin ( UX007 ) in patients with long‐chain fatty acid oxidation disorders: Results from an open‐label , long‐term extension study
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Title
Effects of triheptanoin (
UX007
) in patients with long‐chain fatty acid oxidation disorders: Results from an
open‐label
,
long‐term
extension study
Authors
Keywords
-
Journal
JOURNAL OF INHERITED METABOLIC DISEASE
Volume -, Issue -, Pages -
Publisher
Wiley
Online
2020-09-04
DOI
10.1002/jimd.12313
References
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Related references
Note: Only part of the references are listed.- 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)
- (2019) Jerry Vockley et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Disorders of mitochondrial long-chain fatty acid oxidation and the carnitine shuttle
- (2018) Suzan J. G. Knottnerus et al. REVIEWS IN ENDOCRINE & METABOLIC DISORDERS
- Management and diagnosis of mitochondrial fatty acid oxidation disorders: focus on very-long-chain acyl-CoA dehydrogenase deficiency
- (2018) Kenji Yamada et al. JOURNAL OF HUMAN GENETICS
- UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24 weeks of treatment
- (2017) J. Vockley et al. MOLECULAR GENETICS AND METABOLISM
- Triheptanoin treatment in patients with pediatric cardiomyopathy associated with long chain-fatty acid oxidation disorders
- (2016) J. Vockley et al. MOLECULAR GENETICS AND METABOLISM
- Experiences of caregivers of children with inherited metabolic diseases: a qualitative study
- (2016) Shabnaz Siddiq et al. Orphanet Journal of Rare Diseases
- Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment—A retrospective chart review
- (2015) Jerry Vockley et al. MOLECULAR GENETICS AND METABOLISM
- Anaplerotic treatment of long-chain fat oxidation disorders with triheptanoin: Review of 15years Experience
- (2015) Charles R. Roe et al. MOLECULAR GENETICS AND METABOLISM
- Clinical outcome, biochemical and therapeutic follow-up in 14 Austrian patients with Long-Chain 3-Hydroxy Acyl CoA Dehydrogenase Deficiency (LCHADD)
- (2015) Daniela Karall et al. Orphanet Journal of Rare Diseases
- Blinded Outcome Assessment Was Infrequently Used and Poorly Reported in Open Trials
- (2015) Brennan C. Kahan et al. PLoS One
- Heptanoate as a Neural Fuel: Energetic and Neurotransmitter Precursors in Normal and Glucose Transporter I-Deficient (G1D) Brain
- (2012) Isaac Marin-Valencia et al. JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM
- Clinical and biological features at diagnosis in mitochondrial fatty acid beta-oxidation defects: a French pediatric study of 187 patients
- (2012) Julien Baruteau et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Fatty acid oxidation disorders: outcome and long-term prognosis
- (2010) Bridget Wilcken JOURNAL OF INHERITED METABOLIC DISEASE
- Mitochondrial fatty acid oxidation disorders: clinical presentation of long-chain fatty acid oxidation defects before and after newborn screening
- (2010) Ute Spiekerkoetter JOURNAL OF INHERITED METABOLIC DISEASE
- Parenteral and enteral metabolism of anaplerotic triheptanoin in normal rats. II. Effects on lipolysis, glucose production, and liver acyl-CoA profile
- (2009) Lei Gu et al. AMERICAN JOURNAL OF PHYSIOLOGY-ENDOCRINOLOGY AND METABOLISM
- Monitoring of home safety issues in children on enteral feeds with inherited metabolic disorders
- (2009) S. Evans et al. ARCHIVES OF DISEASE IN CHILDHOOD
- Treatment recommendations in long-chain fatty acid oxidation defects: consensus from a workshop
- (2009) U. Spiekerkoetter et al. JOURNAL OF INHERITED METABOLIC DISEASE
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