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Muscular dystrophy-dystroglycanopathy

MedGen UID:
1842215
Concept ID:
C5679911
Disease or Syndrome
Synonym: Congenital muscular dystrophy due to dystroglycanopathy
 
Monarch Initiative: MONDO:0018276
Orphanet: ORPHA370953

Recent clinical studies

Etiology

Borisovna KO, Yurievna KA, Yurievich TK, Igorevna KO, Olegovich KD, Igorevna DA, Timofeevna BT, Vyacheslavovna ZN, Ivanovna SE, Alekseevich SP, Vladimirovich IV
BMC Pediatr 2019 Apr 8;19(1):98. doi: 10.1186/s12887-019-1470-2. PMID: 30961548Free PMC Article
Peiris TJ, Indaram M, Koo E, Soul JS, Hunter DG
J AAPOS 2018 Jun;22(3):242-244.e1. Epub 2018 Mar 16 doi: 10.1016/j.jaapos.2017.12.011. PMID: 29555514
Kanagawa M, Toda T
J Neuromuscul Dis 2017;4(4):259-267. doi: 10.3233/JND-170255. PMID: 29081423Free PMC Article
Hu P, Wu S, Yuan L, Lin Q, Zheng W, Xia H, Xu H, Guan L, Deng H
J Cell Mol Med 2017 Jul;21(7):1388-1393. Epub 2017 Feb 3 doi: 10.1111/jcmm.13068. PMID: 28157257Free PMC Article
Barone R, Aiello C, Race V, Morava E, Foulquier F, Riemersma M, Passarelli C, Concolino D, Carella M, Santorelli F, Vleugels W, Mercuri E, Garozzo D, Sturiale L, Messina S, Jaeken J, Fiumara A, Wevers RA, Bertini E, Matthijs G, Lefeber DJ
Ann Neurol 2012 Oct;72(4):550-8. doi: 10.1002/ana.23632. PMID: 23109149

Diagnosis

Mohammadi P, Daneshmand MA, Mahdieh N, Ashrafi MR, Heidari M, Garshasbi M
Acta Neurol Belg 2021 Feb;121(1):143-151. Epub 2020 Nov 11 doi: 10.1007/s13760-020-01527-8. PMID: 33175337
Borisovna KO, Yurievna KA, Yurievich TK, Igorevna KO, Olegovich KD, Igorevna DA, Timofeevna BT, Vyacheslavovna ZN, Ivanovna SE, Alekseevich SP, Vladimirovich IV
BMC Pediatr 2019 Apr 8;19(1):98. doi: 10.1186/s12887-019-1470-2. PMID: 30961548Free PMC Article
Hu P, Yuan L, Deng H
Mutat Res Rev Mutat Res 2018 Oct-Dec;778:45-50. Epub 2018 Sep 12 doi: 10.1016/j.mrrev.2018.09.002. PMID: 30454682
Peiris TJ, Indaram M, Koo E, Soul JS, Hunter DG
J AAPOS 2018 Jun;22(3):242-244.e1. Epub 2018 Mar 16 doi: 10.1016/j.jaapos.2017.12.011. PMID: 29555514
Hu P, Wu S, Yuan L, Lin Q, Zheng W, Xia H, Xu H, Guan L, Deng H
J Cell Mol Med 2017 Jul;21(7):1388-1393. Epub 2017 Feb 3 doi: 10.1111/jcmm.13068. PMID: 28157257Free PMC Article

Therapy

Salam MAE, Salama K, Selim YMM, Saad M, Rady R, Alawbathani S, Schroeder S, Elmonem MA, Elkhateeb N
Ann Hum Genet 2023 Jul;87(4):166-173. Epub 2023 Mar 14 doi: 10.1111/ahg.12505. PMID: 36916508
Peiris TJ, Indaram M, Koo E, Soul JS, Hunter DG
J AAPOS 2018 Jun;22(3):242-244.e1. Epub 2018 Mar 16 doi: 10.1016/j.jaapos.2017.12.011. PMID: 29555514
Ducro BJ, Schurink A, Bastiaansen JW, Boegheim IJ, van Steenbeek FG, Vos-Loohuis M, Nijman IJ, Monroe GR, Hellinga I, Dibbits BW, Back W, Leegwater PA
BMC Genomics 2015 Oct 9;16:761. doi: 10.1186/s12864-015-1936-z. PMID: 26452345Free PMC Article

Prognosis

Strang-Karlsson S, Johnson K, Töpf A, Xu L, Lek M, MacArthur DG, Casar-Borota O, Williams M, Straub V, Wallgren-Pettersson C
Neuromuscul Disord 2018 Jul;28(7):614-618. Epub 2018 May 16 doi: 10.1016/j.nmd.2018.04.012. PMID: 29910097Free PMC Article
Hu P, Wu S, Yuan L, Lin Q, Zheng W, Xia H, Xu H, Guan L, Deng H
J Cell Mol Med 2017 Jul;21(7):1388-1393. Epub 2017 Feb 3 doi: 10.1111/jcmm.13068. PMID: 28157257Free PMC Article
Czeschik JC, Hehr U, Hartmann B, Lüdecke HJ, Rosenbaum T, Schweiger B, Wieczorek D
Eur J Med Genet 2013 Dec;56(12):689-94. Epub 2013 Oct 10 doi: 10.1016/j.ejmg.2013.09.014. PMID: 24120487
Barone R, Aiello C, Race V, Morava E, Foulquier F, Riemersma M, Passarelli C, Concolino D, Carella M, Santorelli F, Vleugels W, Mercuri E, Garozzo D, Sturiale L, Messina S, Jaeken J, Fiumara A, Wevers RA, Bertini E, Matthijs G, Lefeber DJ
Ann Neurol 2012 Oct;72(4):550-8. doi: 10.1002/ana.23632. PMID: 23109149

Clinical prediction guides

Bhattacharya S, Das A, Ghosh S, Dasgupta R, Bagchi A
Gene 2014 Mar 1;537(1):108-14. Epub 2013 Dec 18 doi: 10.1016/j.gene.2013.11.071. PMID: 24361964
Barone R, Aiello C, Race V, Morava E, Foulquier F, Riemersma M, Passarelli C, Concolino D, Carella M, Santorelli F, Vleugels W, Mercuri E, Garozzo D, Sturiale L, Messina S, Jaeken J, Fiumara A, Wevers RA, Bertini E, Matthijs G, Lefeber DJ
Ann Neurol 2012 Oct;72(4):550-8. doi: 10.1002/ana.23632. PMID: 23109149

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