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Neurometabolic disease

MedGen UID:
1843198
Concept ID:
C5681332
Disease or Syndrome
Orphanet: ORPHA68385

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVNeurometabolic disease

Professional guidelines

PubMed

Begley D, Gabathuler R, Pastores G, Garcia-Cazorla A, Ardigò D, Scarpa M, Tomanin R, Tosi G
Expert Opin Drug Deliv 2024 Jun;21(6):817-828. Epub 2024 Jul 4 doi: 10.1080/17425247.2024.2375388. PMID: 38963225
Schuurmans IME, Dimitrov B, Schröter J, Ribes A, de la Fuente RP, Zamora B, van Karnebeek CDM, Kölker S, Garanto A
J Inherit Metab Dis 2023 May;46(3):371-390. Epub 2023 Apr 16 doi: 10.1002/jimd.12608. PMID: 37020324
Carlson MD
Curr Opin Neurol 2004 Apr;17(2):133-8. doi: 10.1097/00019052-200404000-00008. PMID: 15021238

Recent clinical studies

Etiology

Mochel F, Gras D, Luton MP, Nizou M, Giovannini D, Delattre C, Aubart M, Barth M, De Saint-Martin A, Doummar D, Essid N, Garros A, Le Camus CH, Hoebeke C, The Tich SN, Perivier M, Rivera S, Rolland A, Roubertie A, Sarret C, Sevin C, Ville D, Sitbon M, Costa JM, Pons R, Garcia-Cazorla A, Vuillaumier S, Petit V, Boespflug-Tanguy O, De Vivo DC; MetaGlut1 Study Group
Neurology 2023 Jun 6;100(23):e2360-e2373. Epub 2023 Apr 19 doi: 10.1212/WNL.0000000000207296. PMID: 37076312Free PMC Article
Fernández-Eulate G, Carreau C, Benoist JF, Lamari F, Rucheton B, Shor N, Nadjar Y
J Neurol Neurosurg Psychiatry 2022 Apr;93(4):413-421. Epub 2022 Feb 9 doi: 10.1136/jnnp-2021-328045. PMID: 35140137Free PMC Article
Carecchio M
Int Rev Neurobiol 2019;149:355-376. Epub 2019 Nov 21 doi: 10.1016/bs.irn.2019.10.009. PMID: 31779820
Haas RH, Parikh S, Falk MJ, Saneto RP, Wolf NI, Darin N, Cohen BH
Pediatrics 2007 Dec;120(6):1326-33. doi: 10.1542/peds.2007-0391. PMID: 18055683
Goebel HH
Brain Dev 1999 Oct;21(7):435-43. doi: 10.1016/s0387-7604(99)00061-3. PMID: 10522518

Diagnosis

Fernández-Eulate G, Carreau C, Benoist JF, Lamari F, Rucheton B, Shor N, Nadjar Y
J Neurol Neurosurg Psychiatry 2022 Apr;93(4):413-421. Epub 2022 Feb 9 doi: 10.1136/jnnp-2021-328045. PMID: 35140137Free PMC Article
Honey MIJ, Jaspers YRJ, Engelen M, Kemp S, Huffnagel IC
Cells 2021 Dec 6;10(12) doi: 10.3390/cells10123427. PMID: 34943935Free PMC Article
Carecchio M
Int Rev Neurobiol 2019;149:355-376. Epub 2019 Nov 21 doi: 10.1016/bs.irn.2019.10.009. PMID: 31779820
Pierre G
Arch Dis Child 2013 Aug;98(8):618-24. Epub 2013 May 22 doi: 10.1136/archdischild-2012-302840. PMID: 23698595
Dhamija R, Patterson MC, Wirrell EC
J Child Neurol 2012 May;27(5):663-71. Epub 2012 Feb 28 doi: 10.1177/0883073811435829. PMID: 22378665

Therapy

Begley D, Gabathuler R, Pastores G, Garcia-Cazorla A, Ardigò D, Scarpa M, Tomanin R, Tosi G
Expert Opin Drug Deliv 2024 Jun;21(6):817-828. Epub 2024 Jul 4 doi: 10.1080/17425247.2024.2375388. PMID: 38963225
Honey MIJ, Jaspers YRJ, Engelen M, Kemp S, Huffnagel IC
Cells 2021 Dec 6;10(12) doi: 10.3390/cells10123427. PMID: 34943935Free PMC Article
Wyse ATS, Grings M, Wajner M, Leipnitz G
Neurotox Res 2019 Feb;35(2):484-494. Epub 2018 Dec 5 doi: 10.1007/s12640-018-9986-z. PMID: 30515714
Ridler C
Nat Rev Neurol 2017 Oct;13(10):576-577. Epub 2017 Aug 29 doi: 10.1038/nrneurol.2017.124. PMID: 28849784
Giannuzzi V, Devlieger H, Margari L, Odlind VL, Ragab L, Bellettato CM, D'Avanzo F, Lampe C, Cassis L, Cortès-Saladelafont E, Cazorla ÁG, Barić I, Cvitanović-Šojat L, Fumić K, Dali CI, Bartoloni F, Bonifazi F, Scarpa M, Ceci A
Eur J Pediatr 2017 Mar;176(3):395-405. Epub 2017 Jan 16 doi: 10.1007/s00431-017-2852-9. PMID: 28093642Free PMC Article

Prognosis

Kemp S, Orsini JJ, Ebberink MS, Engelen M, Lund TC
Mol Genet Metab 2023 Sep-Oct;140(1-2):107678. Epub 2023 Aug 3 doi: 10.1016/j.ymgme.2023.107678. PMID: 37574344
Mochel F, Gras D, Luton MP, Nizou M, Giovannini D, Delattre C, Aubart M, Barth M, De Saint-Martin A, Doummar D, Essid N, Garros A, Le Camus CH, Hoebeke C, The Tich SN, Perivier M, Rivera S, Rolland A, Roubertie A, Sarret C, Sevin C, Ville D, Sitbon M, Costa JM, Pons R, Garcia-Cazorla A, Vuillaumier S, Petit V, Boespflug-Tanguy O, De Vivo DC; MetaGlut1 Study Group
Neurology 2023 Jun 6;100(23):e2360-e2373. Epub 2023 Apr 19 doi: 10.1212/WNL.0000000000207296. PMID: 37076312Free PMC Article
Schuurmans IME, Dimitrov B, Schröter J, Ribes A, de la Fuente RP, Zamora B, van Karnebeek CDM, Kölker S, Garanto A
J Inherit Metab Dis 2023 May;46(3):371-390. Epub 2023 Apr 16 doi: 10.1002/jimd.12608. PMID: 37020324
Honey MIJ, Jaspers YRJ, Engelen M, Kemp S, Huffnagel IC
Cells 2021 Dec 6;10(12) doi: 10.3390/cells10123427. PMID: 34943935Free PMC Article
Carlson MD
Curr Opin Neurol 2004 Apr;17(2):133-8. doi: 10.1097/00019052-200404000-00008. PMID: 15021238

Clinical prediction guides

Martínez-Pizarro A, Picó S, López-Márquez A, Rodriguez-López C, Montalvo E, Alvarez M, Castro M, Ramón-Maiques S, Pérez B, Lucas JJ, Richard E, Desviat LR
Hum Mol Genet 2024 Jun 5;33(12):1074-1089. doi: 10.1093/hmg/ddae051. PMID: 38520741Free PMC Article
Mochel F, Gras D, Luton MP, Nizou M, Giovannini D, Delattre C, Aubart M, Barth M, De Saint-Martin A, Doummar D, Essid N, Garros A, Le Camus CH, Hoebeke C, The Tich SN, Perivier M, Rivera S, Rolland A, Roubertie A, Sarret C, Sevin C, Ville D, Sitbon M, Costa JM, Pons R, Garcia-Cazorla A, Vuillaumier S, Petit V, Boespflug-Tanguy O, De Vivo DC; MetaGlut1 Study Group
Neurology 2023 Jun 6;100(23):e2360-e2373. Epub 2023 Apr 19 doi: 10.1212/WNL.0000000000207296. PMID: 37076312Free PMC Article
Schuurmans IME, Dimitrov B, Schröter J, Ribes A, de la Fuente RP, Zamora B, van Karnebeek CDM, Kölker S, Garanto A
J Inherit Metab Dis 2023 May;46(3):371-390. Epub 2023 Apr 16 doi: 10.1002/jimd.12608. PMID: 37020324
Honey MIJ, Jaspers YRJ, Engelen M, Kemp S, Huffnagel IC
Cells 2021 Dec 6;10(12) doi: 10.3390/cells10123427. PMID: 34943935Free PMC Article
Carlson MD
Curr Opin Neurol 2004 Apr;17(2):133-8. doi: 10.1097/00019052-200404000-00008. PMID: 15021238

Recent systematic reviews

Li JT, Chen ZX, Chen XJ, Jiang YX
Orphanet J Rare Dis 2022 Oct 27;17(1):392. doi: 10.1186/s13023-022-02544-x. PMID: 36303223Free PMC Article
Liu N, Kong XD, Kan QC, Shi HR, Wu QH, Zhuo ZH, Bai QL, Jiang M
J Perinat Med 2016 May 1;44(4):441-51. doi: 10.1515/jpm-2014-0164. PMID: 25431891

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