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Muscle eye brain disease(MEB)

MedGen UID:
105341
Concept ID:
C0457133
Disease or Syndrome
Synonyms: MEB; Santavuori congenital muscular dystrophy
SNOMED CT: Muscle eye brain disease (277950001)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Related gene: POMGNT1
 
Monarch Initiative: MONDO:0018939
Orphanet: ORPHA588

Definition

Congenital muscular dystrophy-dystroglycanopathy with brain and eye anomalies (type A), which includes both the more severe Walker-Warburg syndrome (WWS) and the slightly less severe muscle-eye-brain disease (MEB), is an autosomal recessive disorder with characteristic brain and eye malformations, profound mental retardation, congenital muscular dystrophy, and death usually in the first years of life. It represents the most severe end of a phenotypic spectrum of similar disorders resulting from defective glycosylation of DAG1 (128239), collectively known as 'dystroglycanopathies' (summary by Godfrey et al., 2007). For a general phenotypic description and a discussion of genetic heterogeneity of muscular dystrophy-dystroglycanopathy type A, see MDDGA1 (236670). [from OMIM]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
Follow this link to review classifications for Muscle eye brain disease in Orphanet.

Professional guidelines

PubMed

Geis T, Rödl T, Topaloğlu H, Balci-Hayta B, Hinreiner S, Müller-Felber W, Schoser B, Mehraein Y, Hübner A, Zirn B, Hoopmann M, Reutter H, Mowat D, Schuierer G, Schara U, Hehr U, Kölbel H
Orphanet J Rare Dis 2019 Jul 16;14(1):179. doi: 10.1186/s13023-019-1119-0. PMID: 31311558Free PMC Article
Taniguchi-Ikeda M, Morioka I, Iijima K, Toda T
Mol Aspects Med 2016 Oct;51:115-24. Epub 2016 Jul 12 doi: 10.1016/j.mam.2016.07.003. PMID: 27421908
Balci B, Morris-Rosendahl DJ, Celebi A, Talim B, Topaloglu H, Dinçer P
Prenat Diagn 2007 Jan;27(1):51-4. doi: 10.1002/pd.1622. PMID: 17154333

Recent clinical studies

Etiology

Falsaperla R, Praticò AD, Ruggieri M, Parano E, Rizzo R, Corsello G, Vitaliti G, Pavone P
Ital J Pediatr 2016 Aug 31;42(1):78. doi: 10.1186/s13052-016-0289-9. PMID: 27576556Free PMC Article
Yiş U, Uyanik G, Rosendahl DM, Carman KB, Bayram E, Heise M, Cömertpay G, Kurul SH
Pediatr Neurol 2014 May;50(5):491-7. Epub 2014 Jan 7 doi: 10.1016/j.pediatrneurol.2014.01.008. PMID: 24731844
Voglmeir J, Kaloo S, Laurent N, Meloni MM, Bohlmann L, Wilson IB, Flitsch SL
Biochem J 2011 Jun 1;436(2):447-55. doi: 10.1042/BJ20101059. PMID: 21361872Free PMC Article
Reed UC
Arq Neuropsiquiatr 2009 Jun;67(2A):343-62. doi: 10.1590/s0004-282x2009000200035. PMID: 19547838
Auranen M, Rapola J, Pihko H, Haltia M, Leivo I, Soinila S, Virtanen I, Kalimo H, Anderson LV, Santavuori P, Somer H
Neuromuscul Disord 2000 Jan;10(1):16-23. doi: 10.1016/s0960-8966(99)00066-8. PMID: 10677859

Diagnosis

Arvio M, Määttänen L, Haanpää M, Lähdetie J
Am J Med Genet A 2019 Dec;179(12):2481-2485. Epub 2019 Oct 3 doi: 10.1002/ajmg.a.61369. PMID: 31580529
Falsaperla R, Praticò AD, Ruggieri M, Parano E, Rizzo R, Corsello G, Vitaliti G, Pavone P
Ital J Pediatr 2016 Aug 31;42(1):78. doi: 10.1186/s13052-016-0289-9. PMID: 27576556Free PMC Article
Jiao H, Manya H, Wang S, Zhang Y, Li X, Xiao J, Yang Y, Kobayashi K, Toda T, Endo T, Wu X, Xiong H
Mol Genet Genomics 2013 Aug;288(7-8):297-308. Epub 2013 May 21 doi: 10.1007/s00438-013-0749-5. PMID: 23689641
Shenoy AM, Markowitz JA, Bonnemann CG, Krishnamoorthy K, Bossler AD, Tseng BS
J Clin Neuromuscul Dis 2010 Mar;11(3):124-6. doi: 10.1097/CND.0b013e3181c5054d. PMID: 20215985Free PMC Article
Balci B, Morris-Rosendahl DJ, Celebi A, Talim B, Topaloglu H, Dinçer P
Prenat Diagn 2007 Jan;27(1):51-4. doi: 10.1002/pd.1622. PMID: 17154333

Therapy

AlBalawi AA, Arevalo JF, Abodarahim AM, Alyousif NA, Hazzazi MA
J Pediatr Ophthalmol Strabismus 2023 Mar-Apr;60(2):e8-e10. Epub 2023 Mar 1 doi: 10.3928/01913913-20221229-02. PMID: 36975116
Murphy LB, Schreiber-Katz O, Rafferty K, Robertson A, Topf A, Willis TA, Heidemann M, Thiele S, Bindoff L, Laurent JP, Lochmüller H, Mathews K, Mitchell C, Stevenson JH, Vissing J, Woods L, Walter MC, Straub V
Ann Clin Transl Neurol 2020 May;7(5):757-766. Epub 2020 Apr 28 doi: 10.1002/acn3.51042. PMID: 32342672Free PMC Article
Hackmann T, Skidmore DL, MacManus B
A A Case Rep 2017 Oct 15;9(8):244-247. doi: 10.1213/XAA.0000000000000577. PMID: 28604469
Philpot J, Cowan F, Pennock J, Sewry C, Dubowitz V, Bydder G, Muntoni F
Neuromuscul Disord 1999 Mar;9(2):81-5. doi: 10.1016/s0960-8966(98)00110-2. PMID: 10220862

Prognosis

Traverso M, Baratto S, Iacomino M, Di Duca M, Panicucci C, Casalini S, Grandis M, Falace A, Torella A, Picillo E, Onore ME, Politano L, Nigro V, Innes AM, Barresi R, Bruno C, Zara F, Fiorillo C, Scala M
Eur J Hum Genet 2024 Mar;32(3):342-349. Epub 2024 Jan 4 doi: 10.1038/s41431-023-01516-4. PMID: 38177406Free PMC Article
Voglmeir J, Kaloo S, Laurent N, Meloni MM, Bohlmann L, Wilson IB, Flitsch SL
Biochem J 2011 Jun 1;436(2):447-55. doi: 10.1042/BJ20101059. PMID: 21361872Free PMC Article
Reed UC
Arq Neuropsiquiatr 2009 Jun;67(2A):343-62. doi: 10.1590/s0004-282x2009000200035. PMID: 19547838
Reed UC
Arq Neuropsiquiatr 2009 Mar;67(1):144-68. doi: 10.1590/s0004-282x2009000100038. PMID: 19330236
Zervos A, Hunt KE, Tong HQ, Avallone J, Morales J, Friedman N, Cohen BH, Clark B, Guo S, Gazda H, Beggs AH, Traboulsi EI
Eur J Ophthalmol 2002 Jul-Aug;12(4):253-61. doi: 10.1177/112067210201200401. PMID: 12219993

Clinical prediction guides

Traverso M, Baratto S, Iacomino M, Di Duca M, Panicucci C, Casalini S, Grandis M, Falace A, Torella A, Picillo E, Onore ME, Politano L, Nigro V, Innes AM, Barresi R, Bruno C, Zara F, Fiorillo C, Scala M
Eur J Hum Genet 2024 Mar;32(3):342-349. Epub 2024 Jan 4 doi: 10.1038/s41431-023-01516-4. PMID: 38177406Free PMC Article
Noor SI, Hoffmann M, Rinis N, Bartels MF, Winterhalter PR, Hoelscher C, Hennig R, Himmelreich N, Thiel C, Ruppert T, Rapp E, Strahl S
J Biol Chem 2021 Jan-Jun;296:100433. Epub 2021 Feb 18 doi: 10.1016/j.jbc.2021.100433. PMID: 33610554Free PMC Article
Voglmeir J, Kaloo S, Laurent N, Meloni MM, Bohlmann L, Wilson IB, Flitsch SL
Biochem J 2011 Jun 1;436(2):447-55. doi: 10.1042/BJ20101059. PMID: 21361872Free PMC Article
Taniguchi K, Kobayashi K, Saito K, Yamanouchi H, Ohnuma A, Hayashi YK, Manya H, Jin DK, Lee M, Parano E, Falsaperla R, Pavone P, Van Coster R, Talim B, Steinbrecher A, Straub V, Nishino I, Topaloglu H, Voit T, Endo T, Toda T
Hum Mol Genet 2003 Mar 1;12(5):527-34. doi: 10.1093/hmg/ddg043. PMID: 12588800
Cormand B, Avela K, Pihko H, Santavuori P, Talim B, Topaloglu H, de la Chapelle A, Lehesjoki AE
Am J Hum Genet 1999 Jan;64(1):126-35. doi: 10.1086/302206. PMID: 9915951Free PMC Article

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