Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program-Reference-Cited by-同舟云学术

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Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program

Published:2022-06 Issue:2 Volume:136 Page:152-162
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Vockley Jerry,Enns Gregory M.,Ramirez Antonio Nino,Bedrosian Camille L.,Reineking Bridget,Lu Xiaoxiao,Ray Kathryn,Rahman Syeda,Marsden Deborah

Funder

Ultragenyx Pharmaceutical

NIH

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference28 articles.

1. Triheptanoin treatment in patients with pediatric cardiomyopathy associated with long chain-fatty acid oxidation disorders;Vockley;Mol. Genet. Metab.,2016

2. Long-chain fatty acid oxidation disorders and current management strategies;Vockley;Am. J. Manag. Care,2020

3. Fatty acid oxidation disorders: outcome and long-term prognosis;Wilcken;J. Inherit. Metab. Dis.,2010

4. Clinical manifestations and management of fatty acid oxidation disorders;Merritt;Rev. Endocr. Metab. Disord.,2020

5. Myocardial energy shortage and unmet anaplerotic needs in the fasted long-chain acyl-CoA dehydrogenase knockout mouse;Bakermans;Cardiovasc. Res.,2013

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1. Manifestações clínicas da 3-hidroxiacil-coa desidrogenase de cadeias longas (LCHAD);Caderno Pedagógico;2024-07-29

2. Major clinical events and healthcare resource use among patients with long-chain fatty acid oxidation disorders in the United States: Results from LC-FAOD Odyssey program;Molecular Genetics and Metabolism;2024-05

3. Furosemide/triheptanoin;Reactions Weekly;2023-07-01

4. Clinical, biochemical and molecular characterization of 12 patients with pyruvate carboxylase deficiency treated with triheptanoin;Molecular Genetics and Metabolism;2023-06

5. Peroxisome proliferator-activated receptor (PPAR) agonists as a potential therapy for inherited metabolic disorders;Biochemical Pharmacology;2023-03

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