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• W3090469015 startingPage "e699" @default.
• W3090469015 abstract "Inborn errors of metabolism (IEMs) are inherited defects in a metabolic pathway resulting in clinical disease. The overall goal of therapy is to restore metabolic homeostasis while minimizing the deleterious effects of the interruption. Conventional treatments focus on decreasing substrate, providing product, and replacing deficient enzyme or cofactor. We discuss examples of established, novel, and emerging therapies to provide a framework for understanding the principles of management for patients with IEMs." @default.
• W3090469015 created "2020-10-08" @default.
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• W3090469015 date "2020-10-01" @default.
• W3090469015 modified "2023-09-23" @default.
• W3090469015 title "Established and Emerging Treatments for Patients with Inborn Errors of Metabolism" @default.
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• W3090469015 doi "https://doi.org/10.1542/neo.21-10-e699" @default.
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