Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program
Published 2022 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program
Authors
Keywords
Fatty acid oxidation disorder, LC-FAOD, Triheptanoin
Journal
MOLECULAR GENETICS AND METABOLISM
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2022-04-12
DOI
10.1016/j.ymgme.2022.04.001
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Long-term experience with triheptanoin in 12 Austrian patients with long-chain fatty acid oxidation disorders
- (2021) Thomas Zöggeler et al. Orphanet Journal of Rare Diseases
- Clinical outcomes in a series of 18 patients with long chain fatty acids oxidation disorders treated with triheptanoin for a median duration of 22 months
- (2021) Nathalie Guffon et al. MOLECULAR GENETICS AND METABOLISM
- Clinical manifestations and management of fatty acid oxidation disorders
- (2020) J. Lawrence Merritt et al. REVIEWS IN ENDOCRINE & METABOLIC DISORDERS
- Triheptanoin: First Approval
- (2020) Matt Shirley DRUGS
- Effects of triheptanoin ( UX007 ) in patients with long‐chain fatty acid oxidation disorders: Results from an open‐label , long‐term extension study
- (2020) Jerry Vockley et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Nutrition management guideline for very-long chain acyl-CoA dehydrogenase deficiency (VLCAD): An evidence- and consensus-based approach
- (2020) S.C. Van Calcar et al. MOLECULAR GENETICS AND METABOLISM
- 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
- Triheptanoin treatment in patients with pediatric cardiomyopathy associated with long chain-fatty acid oxidation disorders
- (2016) J. Vockley et al. MOLECULAR GENETICS AND METABOLISM
- 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
- Development and pathomechanisms of cardiomyopathy in very long-chain acyl-CoA dehydrogenase deficient (VLCAD−/−) mice
- (2014) Sara Tucci et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Myocardial energy shortage and unmet anaplerotic needs in the fasted long-chain acyl-CoA dehydrogenase knockout mouse
- (2013) Adrianus J. Bakermans et al. CARDIOVASCULAR RESEARCH
- 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
- Modulating fatty acid oxidation in heart failure
- (2011) V. Lionetti et al. CARDIOVASCULAR RESEARCH
- Efficacy and outcome of expanded newborn screening for metabolic diseases - Report of 10 years from South-West Germany *
- (2011) Martin Lindner et al. Orphanet Journal of Rare Diseases
- Fatty acid oxidation disorders: outcome and long-term prognosis
- (2010) Bridget Wilcken JOURNAL OF INHERITED METABOLIC DISEASE
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now