Inborn Errors of Metabolism - PubMed (original) (raw)

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In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan.

2023 Jul 17.

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Inborn Errors of Metabolism

Rebecca Jeanmonod et al.

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Inborn errors of metabolism are a heterogeneous group of disorders that may be inherited or may occur as the result of spontaneous mutation. These diseases involve failure of the metabolic pathways involved in either the break-down or storage of carbohydrates, fatty acids, and proteins. Although any given inborn error of metabolism is very rare, taken as a group, inborn errors occur in 1 in 2500 births, making them quite common. They can present at any age, and therefore, a working knowledge of these diseases, their presentations, and their evaluation is critical for the emergency provider.

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Conflict of interest statement

Disclosure: Rebecca Jeanmonod declares no relevant financial relationships with ineligible companies.

Disclosure: Edinen Asuka declares no relevant financial relationships with ineligible companies.

Disclosure: Donald Jeanmonod declares no relevant financial relationships with ineligible companies.

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References

    1. Stenton SL, Kremer LS, Kopajtich R, Ludwig C, Prokisch H. The diagnosis of inborn errors of metabolism by an integrative "multi-omics" approach: A perspective encompassing genomics, transcriptomics, and proteomics. J Inherit Metab Dis. 2020 Jan;43(1):25-35. - PubMed
    1. Marques EP, Wyse ATS. Creatine as a Neuroprotector: an Actor that Can Play Many Parts. Neurotox Res. 2019 Aug;36(2):411-423. - PubMed
    1. Guerrero RB, Kloke KM, Salazar D. Inborn Errors of Metabolism and the Gastrointestinal Tract. Gastroenterol Clin North Am. 2019 Jun;48(2):183-198. - PubMed
    1. Canton M, Gall DL, Feillet F, Bonnemains C, Roy A. Neuropsychological Profile of Children with Early and Continuously Treated Phenylketonuria: Systematic Review and Future Approaches. J Int Neuropsychol Soc. 2019 Jul;25(6):624-643. - PubMed
    1. Ferreira CR, Cassiman D, Blau N. Clinical and biochemical footprints of inherited metabolic diseases. II. Metabolic liver diseases. Mol Genet Metab. 2019 Jun;127(2):117-121. - PMC - PubMed

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