Dietary management and major clinical events in patients with long-chain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study
Author:
Funder
Ultragenyx Pharmaceutical
Publisher
Elsevier BV
Subject
Nutrition and Dietetics,Endocrinology, Diabetes and Metabolism
Reference13 articles.
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2. Management and outcome in 75 individuals with long-chain fatty acid oxidation defects: results from a workshop;Spiekerkoetter;J Inherit Metab Dis,2009
3. Efficacy and outcome of expanded newborn screening for metabolic diseases - report of 10 years from South-West Germany;Lindner;Orphanet J Rare Dis,2011
4. Anaplerotic treatment of long-chain fat oxidation disorders with triheptanoin: review of 15 years experience;Roe;Mol Genet Metabol,2015
5. Treatment recommendations in long-chain fatty acid oxidation defects: consensus from a workshop;Spiekerkoetter;J Inherit Metab Dis,2009
Cited by 4 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Challenges and strategies for clinical trials in propionic and methylmalonic acidemias;Molecular Genetics and Metabolism;2023-07
2. Triheptanoin for the treatment of long‐chain fatty acid oxidation disorders: Final results of an open‐label, long‐term extension study;Journal of Inherited Metabolic Disease;2023-06-19
3. The Pharmacokinetics of Triheptanoin and Its Metabolites in Healthy Subjects and Patients With Long‐Chain Fatty Acid Oxidation Disorders;Clinical Pharmacology in Drug Development;2021-03-31
4. Diagnosis and Clinical Management of Long-chain Fatty-acid Oxidation Disorders: A Review;European Endocrinology;2021
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