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Inborn error of amino acid metabolism

MedGen UID:
1867
Concept ID:
C0002514
Disease or Syndrome
Synonyms: Amino Acid Metabolism Disorders, Inborn; Amino Acid Metabolism, Inborn Error; Amino Acid Metabolism, Inborn Errors; Amino Acidopathies, Congenital; Amino Acidopathies, Inborn; Amino Acidopathy, Congenital; Amino Acidopathy, Inborn; Congenital Amino Acidopathies; Congenital Amino Acidopathy; Inborn Amino Acidopathies; Inborn Amino Acidopathy; Inborn Errors, Amino Acid Metabolism
SNOMED CT: Inborn error of amino acid metabolism (42930003); Hyperaminoaciduria (42930003)

Definition

An inherited disorder that affects the metabolism of the amino acids. Representative examples include alkaptonuria, homocystinuria, tyrosinemia, and phenylketonuria. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVInborn error of amino acid metabolism

Professional guidelines

PubMed

Balfoort BM, Buijs MJN, Ten Asbroek ALMA, Bergen AAB, Boon CJF, Ferreira EA, Houtkooper RH, Wagenmakers MAEM, Wanders RJA, Waterham HR, Timmer C, van Karnebeek CD, Brands MM
Mol Genet Metab 2021 Sep-Oct;134(1-2):96-116. Epub 2021 Jul 26 doi: 10.1016/j.ymgme.2021.07.010. PMID: 34340878
Ferreira F, Azevedo L, Neiva R, Sousa C, Fonseca H, Marcão A, Rocha H, Carmona C, Ramos S, Bandeira A, Martins E, Campos T, Rodrigues E, Garcia P, Diogo L, Ferreira AC, Sequeira S, Silva F, Rodrigues L, Gaspar A, Janeiro P, Amorim A, Vilarinho L
Mol Genet Genomic Med 2021 Mar;9(3):e1559. Epub 2021 Jan 19 doi: 10.1002/mgg3.1559. PMID: 33465300Free PMC Article
Tresbach RH, Sperb-Ludwig F, Ligabue-Braun R, Tonon T, de Oliveira Cardoso MT, Heredia RS, da Silva Rosa MTA, Martins BC, Poubel MO, da Silva LCS, Maillot F, Schwartz IVD
Genes (Basel) 2020 Dec 25;12(1) doi: 10.3390/genes12010020. PMID: 33375644Free PMC Article

Recent clinical studies

Etiology

de Castro MJ, de Lamas C, Sánchez-Pintos P, González-Lamuño D, Couce ML
Nutrients 2020 Jul 20;12(7) doi: 10.3390/nu12072154. PMID: 32698408Free PMC Article
Guerra IMS, Ferreira HB, Neves B, Melo T, Diogo LM, Domingues MR, Moreira ASP
Arch Biochem Biophys 2020 Jul 30;688:108431. Epub 2020 May 24 doi: 10.1016/j.abb.2020.108431. PMID: 32461102
Couce ML, de Castro MJ, de Lamas C, Leis R
Nutrients 2019 Jul 6;11(7) doi: 10.3390/nu11071537. PMID: 31284588Free PMC Article
Shirzadeh T, Saeidian AH, Bagherian H, Salehpour S, Setoodeh A, Alaei MR, Youssefian L, Samavat A, Touati A, Fallah MS, Vahidnezhad H, Karimipoor M, Azadmehr S, Raeisi M, Bandehi Sarhadi A, Zafarghandi Motlagh F, Jamali M, Zeinali Z, Abiri M, Zeinali S; Additional individual contributors
J Inherit Metab Dis 2018 Nov;41(6):1159-1167. Epub 2018 Aug 29 doi: 10.1007/s10545-018-0228-6. PMID: 30159852
Rocha JC, Martins MJ
J Inherit Metab Dis 2012 May;35(3):381-98. Epub 2011 Nov 25 doi: 10.1007/s10545-011-9417-2. PMID: 22116469

Diagnosis

Guerra IMS, Ferreira HB, Neves B, Melo T, Diogo LM, Domingues MR, Moreira ASP
Arch Biochem Biophys 2020 Jul 30;688:108431. Epub 2020 May 24 doi: 10.1016/j.abb.2020.108431. PMID: 32461102
Couce ML, de Castro MJ, de Lamas C, Leis R
Nutrients 2019 Jul 6;11(7) doi: 10.3390/nu11071537. PMID: 31284588Free PMC Article
Maguire CA, Chong H, Ramachandran R, Popoola J, Akhras V, Singh M
Clin Exp Dermatol 2018 Apr;43(3):315-318. Epub 2018 Jan 15 doi: 10.1111/ced.13369. PMID: 29336047
Rocha JC, Martins MJ
J Inherit Metab Dis 2012 May;35(3):381-98. Epub 2011 Nov 25 doi: 10.1007/s10545-011-9417-2. PMID: 22116469
Gadian DG, Proctor E, Williams SR, Cady EB, Gardiner RM
Magn Reson Med 1986 Feb;3(1):150-6. doi: 10.1002/mrm.1910030123. PMID: 3959882

Therapy

Delbreil P, Dhondt S, Kenaan El Rahbani RM, Banquy X, Mitchell JJ, Brambilla D
Adv Healthc Mater 2024 Oct;13(26):e2401353. Epub 2024 May 31 doi: 10.1002/adhm.202401353. PMID: 38801163
Balfoort BM, Buijs MJN, Ten Asbroek ALMA, Bergen AAB, Boon CJF, Ferreira EA, Houtkooper RH, Wagenmakers MAEM, Wanders RJA, Waterham HR, Timmer C, van Karnebeek CD, Brands MM
Mol Genet Metab 2021 Sep-Oct;134(1-2):96-116. Epub 2021 Jul 26 doi: 10.1016/j.ymgme.2021.07.010. PMID: 34340878
de Castro MJ, de Lamas C, Sánchez-Pintos P, González-Lamuño D, Couce ML
Nutrients 2020 Jul 20;12(7) doi: 10.3390/nu12072154. PMID: 32698408Free PMC Article
Couce ML, de Castro MJ, de Lamas C, Leis R
Nutrients 2019 Jul 6;11(7) doi: 10.3390/nu11071537. PMID: 31284588Free PMC Article
Jaffe EK
Mol Genet Metab 2017 Aug;121(4):289-296. Epub 2017 Jun 15 doi: 10.1016/j.ymgme.2017.06.005. PMID: 28645531Free PMC Article

Prognosis

Tresbach RH, Sperb-Ludwig F, Ligabue-Braun R, Tonon T, de Oliveira Cardoso MT, Heredia RS, da Silva Rosa MTA, Martins BC, Poubel MO, da Silva LCS, Maillot F, Schwartz IVD
Genes (Basel) 2020 Dec 25;12(1) doi: 10.3390/genes12010020. PMID: 33375644Free PMC Article
Guerra IMS, Ferreira HB, Neves B, Melo T, Diogo LM, Domingues MR, Moreira ASP
Arch Biochem Biophys 2020 Jul 30;688:108431. Epub 2020 May 24 doi: 10.1016/j.abb.2020.108431. PMID: 32461102
Aldámiz-Echevarría L, Llarena M, Bueno MA, Dalmau J, Vitoria I, Fernández-Marmiesse A, Andrade F, Blasco J, Alcalde C, Gil D, García MC, González-Lamuño D, Ruiz M, Ruiz MA, Peña-Quintana L, González D, Sánchez-Valverde F, Desviat LR, Pérez B, Couce ML
J Hum Genet 2016 Aug;61(8):731-44. Epub 2016 Apr 28 doi: 10.1038/jhg.2016.38. PMID: 27121329
Rocha JC, Martins MJ
J Inherit Metab Dis 2012 May;35(3):381-98. Epub 2011 Nov 25 doi: 10.1007/s10545-011-9417-2. PMID: 22116469
Scheinman JI
Miner Electrolyte Metab 1994;20(6):340-51. PMID: 7783696

Clinical prediction guides

Tresbach RH, Sperb-Ludwig F, Ligabue-Braun R, Tonon T, de Oliveira Cardoso MT, Heredia RS, da Silva Rosa MTA, Martins BC, Poubel MO, da Silva LCS, Maillot F, Schwartz IVD
Genes (Basel) 2020 Dec 25;12(1) doi: 10.3390/genes12010020. PMID: 33375644Free PMC Article
de Castro MJ, de Lamas C, Sánchez-Pintos P, González-Lamuño D, Couce ML
Nutrients 2020 Jul 20;12(7) doi: 10.3390/nu12072154. PMID: 32698408Free PMC Article
Guerra IMS, Ferreira HB, Neves B, Melo T, Diogo LM, Domingues MR, Moreira ASP
Arch Biochem Biophys 2020 Jul 30;688:108431. Epub 2020 May 24 doi: 10.1016/j.abb.2020.108431. PMID: 32461102
Faverzani JL, Hammerschmidt TG, Sitta A, Deon M, Wajner M, Vargas CR
Cell Mol Neurobiol 2017 Nov;37(8):1477-1485. Epub 2017 Mar 3 doi: 10.1007/s10571-017-0478-0. PMID: 28258516
Cazzorla C, Cegolon L, Burlina AP, Celato A, Massa P, Giordano L, Polo G, Daniele A, Salvatore F, Burlina AB
BMC Public Health 2014 Dec 4;14:1243. doi: 10.1186/1471-2458-14-1243. PMID: 25471331Free PMC Article

Recent systematic reviews

de Castro MJ, de Lamas C, Sánchez-Pintos P, González-Lamuño D, Couce ML
Nutrients 2020 Jul 20;12(7) doi: 10.3390/nu12072154. PMID: 32698408Free PMC Article
Couce ML, de Castro MJ, de Lamas C, Leis R
Nutrients 2019 Jul 6;11(7) doi: 10.3390/nu11071537. PMID: 31284588Free PMC Article

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