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Disorder of the urea cycle metabolism(UCD)

MedGen UID:
57586
Concept ID:
C0154246
Disease or Syndrome
Synonyms: UCD; Urea cycle disorder; Urea Cycle Disorders
SNOMED CT: Disorder of urea cycle (36444000); Disorder of the urea cycle metabolism (36444000)
 
Related genes: NAGS, OTC, CPS1, ASS1, ASL, ARG1
 
Monarch Initiative: MONDO:0004739
Orphanet: ORPHA79167

Definition

A genetic inborn error of metabolism characterized by the deficiency of one of the enzymes necessary for the urea cycle. It results in accumulation of ammonia in the body. [from NCI]

Professional guidelines

PubMed

Häberle J, Burlina A, Chakrapani A, Dixon M, Karall D, Lindner M, Mandel H, Martinelli D, Pintos-Morell G, Santer R, Skouma A, Servais A, Tal G, Rubio V, Huemer M, Dionisi-Vici C
J Inherit Metab Dis 2019 Nov;42(6):1192-1230. Epub 2019 May 15 doi: 10.1002/jimd.12100. PMID: 30982989
Auron A, Brophy PD
Pediatr Nephrol 2012 Feb;27(2):207-22. Epub 2011 Mar 23 doi: 10.1007/s00467-011-1838-5. PMID: 21431427
Häberle J
Eur J Pediatr 2011 Jan;170(1):21-34. Epub 2010 Dec 17 doi: 10.1007/s00431-010-1369-2. PMID: 21165747

Recent clinical studies

Etiology

Komatsu M, Tanaka N, Kimura T, Yazaki M
Nutrients 2023 May 12;15(10) doi: 10.3390/nu15102284. PMID: 37242166Free PMC Article
Bousman CA, Bengesser SA, Aitchison KJ, Amare AT, Aschauer H, Baune BT, Asl BB, Bishop JR, Burmeister M, Chaumette B, Chen LS, Cordner ZA, Deckert J, Degenhardt F, DeLisi LE, Folkersen L, Kennedy JL, Klein TE, McClay JL, McMahon FJ, Musil R, Saccone NL, Sangkuhl K, Stowe RM, Tan EC, Tiwari AK, Zai CC, Zai G, Zhang J, Gaedigk A, Müller DJ
Pharmacopsychiatry 2021 Jan;54(1):5-17. Epub 2020 Nov 4 doi: 10.1055/a-1288-1061. PMID: 33147643
Zielonka M, Kölker S, Gleich F, Stützenberger N, Nagamani SCS, Gropman AL, Hoffmann GF, Garbade SF, Posset R; Urea Cycle Disorders Consortium (UCDC) and the European Registry and Network for Intoxication type Metabolic Diseases (E-IMD) Consortia Study Group
Ann Clin Transl Neurol 2019 Sep;6(9):1858-1871. Epub 2019 Aug 30 doi: 10.1002/acn3.50886. PMID: 31469252Free PMC Article
Pearson TS, Pons R, Ghaoui R, Sue CM
Mov Disord 2019 May;34(5):625-636. Epub 2019 Mar 26 doi: 10.1002/mds.27655. PMID: 30913345
Summar ML, Mew NA
Pediatr Clin North Am 2018 Apr;65(2):231-246. Epub 2018 Feb 2 doi: 10.1016/j.pcl.2017.11.004. PMID: 29502911

Diagnosis

Komatsu M, Tanaka N, Kimura T, Yazaki M
Nutrients 2023 May 12;15(10) doi: 10.3390/nu15102284. PMID: 37242166Free PMC Article
Häberle J, Burlina A, Chakrapani A, Dixon M, Karall D, Lindner M, Mandel H, Martinelli D, Pintos-Morell G, Santer R, Skouma A, Servais A, Tal G, Rubio V, Huemer M, Dionisi-Vici C
J Inherit Metab Dis 2019 Nov;42(6):1192-1230. Epub 2019 May 15 doi: 10.1002/jimd.12100. PMID: 30982989
Summar ML, Mew NA
Pediatr Clin North Am 2018 Apr;65(2):231-246. Epub 2018 Feb 2 doi: 10.1016/j.pcl.2017.11.004. PMID: 29502911
Häberle J, Boddaert N, Burlina A, Chakrapani A, Dixon M, Huemer M, Karall D, Martinelli D, Crespo PS, Santer R, Servais A, Valayannopoulos V, Lindner M, Rubio V, Dionisi-Vici C
Orphanet J Rare Dis 2012 May 29;7:32. doi: 10.1186/1750-1172-7-32. PMID: 22642880Free PMC Article
Auron A, Brophy PD
Pediatr Nephrol 2012 Feb;27(2):207-22. Epub 2011 Mar 23 doi: 10.1007/s00467-011-1838-5. PMID: 21431427

Therapy

Shakerdi L, Ryan A
J Clin Pathol 2023 Aug;76(8):501-509. Epub 2023 May 10 doi: 10.1136/jcp-2022-208644. PMID: 37164630
Bousman CA, Bengesser SA, Aitchison KJ, Amare AT, Aschauer H, Baune BT, Asl BB, Bishop JR, Burmeister M, Chaumette B, Chen LS, Cordner ZA, Deckert J, Degenhardt F, DeLisi LE, Folkersen L, Kennedy JL, Klein TE, McClay JL, McMahon FJ, Musil R, Saccone NL, Sangkuhl K, Stowe RM, Tan EC, Tiwari AK, Zai CC, Zai G, Zhang J, Gaedigk A, Müller DJ
Pharmacopsychiatry 2021 Jan;54(1):5-17. Epub 2020 Nov 4 doi: 10.1055/a-1288-1061. PMID: 33147643
Raina R, Bedoyan JK, Lichter-Konecki U, Jouvet P, Picca S, Mew NA, Machado MC, Chakraborty R, Vemuganti M, Grewal MK, Bunchman T, Sethi SK, Krishnappa V, McCulloch M, Alhasan K, Bagga A, Basu RK, Schaefer F, Filler G, Warady BA
Nat Rev Nephrol 2020 Aug;16(8):471-482. Epub 2020 Apr 8 doi: 10.1038/s41581-020-0267-8. PMID: 32269302Free PMC Article
Häberle J, Boddaert N, Burlina A, Chakrapani A, Dixon M, Huemer M, Karall D, Martinelli D, Crespo PS, Santer R, Servais A, Valayannopoulos V, Lindner M, Rubio V, Dionisi-Vici C
Orphanet J Rare Dis 2012 May 29;7:32. doi: 10.1186/1750-1172-7-32. PMID: 22642880Free PMC Article
Wilson JM
Mol Genet Metab 2009 Apr;96(4):151-7. Epub 2009 Feb 10 doi: 10.1016/j.ymgme.2008.12.016. PMID: 19211285

Prognosis

Tavoulari S, Lacabanne D, Thangaratnarajah C, Kunji ERS
Trends Endocrinol Metab 2022 Aug;33(8):539-553. Epub 2022 Jun 17 doi: 10.1016/j.tem.2022.05.002. PMID: 35725541Free PMC Article
Zielonka M, Kölker S, Gleich F, Stützenberger N, Nagamani SCS, Gropman AL, Hoffmann GF, Garbade SF, Posset R; Urea Cycle Disorders Consortium (UCDC) and the European Registry and Network for Intoxication type Metabolic Diseases (E-IMD) Consortia Study Group
Ann Clin Transl Neurol 2019 Sep;6(9):1858-1871. Epub 2019 Aug 30 doi: 10.1002/acn3.50886. PMID: 31469252Free PMC Article
Matsumoto S, Häberle J, Kido J, Mitsubuchi H, Endo F, Nakamura K
J Hum Genet 2019 Sep;64(9):833-847. Epub 2019 May 20 doi: 10.1038/s10038-019-0614-4. PMID: 31110235
Pearson TS, Pons R, Ghaoui R, Sue CM
Mov Disord 2019 May;34(5):625-636. Epub 2019 Mar 26 doi: 10.1002/mds.27655. PMID: 30913345
Häberle J
Eur J Pediatr 2011 Jan;170(1):21-34. Epub 2010 Dec 17 doi: 10.1007/s00431-010-1369-2. PMID: 21165747

Clinical prediction guides

Lo RS, Cromie GA, Tang M, Teng K, Owens K, Sirr A, Kutz JN, Morizono H, Caldovic L, Ah Mew N, Gropman A, Dudley AM
Am J Hum Genet 2023 May 4;110(5):863-879. doi: 10.1016/j.ajhg.2023.03.019. PMID: 37146589Free PMC Article
Tavoulari S, Lacabanne D, Thangaratnarajah C, Kunji ERS
Trends Endocrinol Metab 2022 Aug;33(8):539-553. Epub 2022 Jun 17 doi: 10.1016/j.tem.2022.05.002. PMID: 35725541Free PMC Article
Zielonka M, Kölker S, Gleich F, Stützenberger N, Nagamani SCS, Gropman AL, Hoffmann GF, Garbade SF, Posset R; Urea Cycle Disorders Consortium (UCDC) and the European Registry and Network for Intoxication type Metabolic Diseases (E-IMD) Consortia Study Group
Ann Clin Transl Neurol 2019 Sep;6(9):1858-1871. Epub 2019 Aug 30 doi: 10.1002/acn3.50886. PMID: 31469252Free PMC Article
Häberle J, Boddaert N, Burlina A, Chakrapani A, Dixon M, Huemer M, Karall D, Martinelli D, Crespo PS, Santer R, Servais A, Valayannopoulos V, Lindner M, Rubio V, Dionisi-Vici C
Orphanet J Rare Dis 2012 May 29;7:32. doi: 10.1186/1750-1172-7-32. PMID: 22642880Free PMC Article
Grünert SC, Stucki M, Morscher RJ, Suormala T, Bürer C, Burda P, Christensen E, Ficicioglu C, Herwig J, Kölker S, Möslinger D, Pasquini E, Santer R, Schwab KO, Wilcken B, Fowler B, Yue WW, Baumgartner MR
Orphanet J Rare Dis 2012 May 29;7:31. doi: 10.1186/1750-1172-7-31. PMID: 22642865Free PMC Article

Recent systematic reviews

Bin Sawad A, Jackimiec J, Bechter M, Trucillo A, Lindsley K, Bhagat A, Uyei J, Diaz GA
Mol Genet Metab 2022 Sep-Oct;137(1-2):153-163. Epub 2022 Aug 25 doi: 10.1016/j.ymgme.2022.08.005. PMID: 36049366
Bousman CA, Bengesser SA, Aitchison KJ, Amare AT, Aschauer H, Baune BT, Asl BB, Bishop JR, Burmeister M, Chaumette B, Chen LS, Cordner ZA, Deckert J, Degenhardt F, DeLisi LE, Folkersen L, Kennedy JL, Klein TE, McClay JL, McMahon FJ, Musil R, Saccone NL, Sangkuhl K, Stowe RM, Tan EC, Tiwari AK, Zai CC, Zai G, Zhang J, Gaedigk A, Müller DJ
Pharmacopsychiatry 2021 Jan;54(1):5-17. Epub 2020 Nov 4 doi: 10.1055/a-1288-1061. PMID: 33147643
Kenneson A, Singh RH
Orphanet J Rare Dis 2020 Oct 9;15(1):279. doi: 10.1186/s13023-020-01560-z. PMID: 33036647Free PMC Article
Martinelli D, Diodato D, Ponzi E, Monné M, Boenzi S, Bertini E, Fiermonte G, Dionisi-Vici C
Orphanet J Rare Dis 2015 Mar 11;10:29. doi: 10.1186/s13023-015-0242-9. PMID: 25874378Free PMC Article
van Karnebeek CD, Stockler S
Mol Genet Metab 2012 Mar;105(3):368-81. Epub 2011 Nov 30 doi: 10.1016/j.ymgme.2011.11.191. PMID: 22212131

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