U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

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
Tarailo-Graovac M, Shyr C, Ross CJ, Horvath GA, Salvarinova R, Ye XC, Zhang LH, Bhavsar AP, Lee JJ, Drögemöller BI, Abdelsayed M, Alfadhel M, Armstrong L, Baumgartner MR, Burda P, Connolly MB, Cameron J, Demos M, Dewan T, Dionne J, Evans AM, Friedman JM, Garber I, Lewis S, Ling J, Mandal R, Mattman A, McKinnon M, Michoulas A, Metzger D, Ogunbayo OA, Rakic B, Rozmus J, Ruben P, Sayson B, Santra S, Schultz KR, Selby K, Shekel P, Sirrs S, Skrypnyk C, Superti-Furga A, Turvey SE, Van Allen MI, Wishart D, Wu J, Wu J, Zafeiriou D, Kluijtmans L, Wevers RA, Eydoux P, Lehman AM, Vallance H, Stockler-Ipsiroglu S, Sinclair G, Wasserman WW, van Karnebeek CD
N Engl J Med 2016 Jun 9;374(23):2246-55. Epub 2016 May 25 doi: 10.1056/NEJMoa1515792. PMID: 27276562Free PMC Article

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
Hagberg B
Eur J Pediatr 1990 Feb;149(5):296-305. doi: 10.1007/BF02171552. PMID: 2155788

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
Filiano JJ
Clin Perinatol 2006 Jun;33(2):411-79. doi: 10.1016/j.clp.2006.03.013. PMID: 16765732

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
Rivera-Barahona A, Fulgencio-Covián A, Pérez-Cerdá C, Ramos R, Barry MA, Ugarte M, Pérez B, Richard E, Desviat LR
Sci Rep 2017 Jul 18;7(1):5727. doi: 10.1038/s41598-017-06420-8. PMID: 28720782Free PMC Article

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
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
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
Rivera-Barahona A, Fulgencio-Covián A, Pérez-Cerdá C, Ramos R, Barry MA, Ugarte M, Pérez B, Richard E, Desviat LR
Sci Rep 2017 Jul 18;7(1):5727. doi: 10.1038/s41598-017-06420-8. PMID: 28720782Free PMC Article

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

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Consumer resources

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...