P013: A long-term disease monitoring program in patients with long-chain fatty acid oxidation disorders
Inbar-Feigenberg, Michal, Burton, Barbara, Sanchez-Valle, Amarilis, Vockley, Jerry, Aggarwal, Anjali, Hetzer, Joel, Reineking, Bridget, Grimm, Andrew
Published in Genetics in Medicine Open (2024)
Published in Genetics in Medicine Open (2024)
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Journal Article
Early results from a long-term disease monitoring program in patients with long-chain fatty acid oxidation disorders
Sanchez-Valle, Amarilis, Vockley, Jerry, Burton, Barbara, Inbar-Feigenberg, Michal, Aggarwal, Anjali, Hetzer, Joel, Reineking, Bridget, Grimm, Andrew
Published in Molecular genetics and metabolism (01.04.2024)
Published in Molecular genetics and metabolism (01.04.2024)
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eP495: Introduction to Odyssey: Real-world rare disease data collection program for patients with long-chain fatty acid oxidation disorders in the US
Kruger, Eliza, Yang, Erru, Reineking, Bridget, Thomas, Nina, Mace, Kieran, Tierney, Meghan, Cibelli, Emily, Drozd, Dan, Ross, Nathan, Kritzer, Amy
Published in Genetics in medicine (01.03.2022)
Published in Genetics in medicine (01.03.2022)
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Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program
Vockley, Jerry, Enns, Gregory M., Ramirez, Antonio Nino, Bedrosian, Camille L., Reineking, Bridget, Lu, Xiaoxiao, Ray, Kathryn, Rahman, Syeda, Marsden, Deborah
Published in Molecular genetics and metabolism (01.06.2022)
Published in Molecular genetics and metabolism (01.06.2022)
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Journal Article
Clinical outcomes of major clinical events and emergency triheptanoin use in critically ill patients with long-chain fatty acid oxidation disorders
Enns, Gregory, Marsden, Deborah, Ramirez, Antonio Nino, Bedrosian, Camille, Reineking, Bridget, Lu, Xiaoxiao, Rahman, Syeda, Vockley, Jerry
Published in Molecular genetics and metabolism (01.04.2021)
Published in Molecular genetics and metabolism (01.04.2021)
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Journal Article
eP130 - Clinical outcomes of major clinical events and emergency triheptanoin use in critically ill patients with long-chain fatty acid oxidation disorders
Enns, Gregory, Marsden, Deborah, Ramirez, Antonio Nino, Bedrosian, Camille, Reineking, Bridget, Lu, Xiaoxiao, Rahman, Syeda, Vockley, Jerry
Published in Molecular genetics and metabolism (01.04.2021)
Published in Molecular genetics and metabolism (01.04.2021)
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Journal Article
Triheptanoin for the treatment of long-chain fatty acid oxidation disorders: Final results of an open-label, long-term extension study
Vockley, Jerry, Burton, Barbara K, Berry, Gerard, Longo, Nicola, Phillips, John, Sanchez-Valle, Amarilis, Chapman, Kimberly A, Tanpaiboon, Pranoot, Grunewald, Stephanie, Murphy, Elaine, Lu, Xiaoxiao, Rahman, Syeda, Ray, Kathryn, Reineking, Bridget, Pisani, Laura, Ramirez, Antonio Nino
Published in Journal of inherited metabolic disease (01.09.2023)
Published in Journal of inherited metabolic disease (01.09.2023)
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Journal Article