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Galloway-Mowat syndrome(CAMOS)

MedGen UID:
167086
Concept ID:
C0795949
Disease or Syndrome
Synonyms: GALLOWAY SYNDROME; Microcephaly nephrosis syndrome; Microcephaly, hiatal hernia and nephrotic syndrome; Nephrosis neuronal dysmigration syndrome
SNOMED CT: Galloway Mowat syndrome (721297008); Galloway syndrome (721297008); Microcephaly, hiatus hernia, nephrotic syndrome (721297008); Nephrosis, neuronal dysmigration syndrome (721297008)
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).
X-linked recessive inheritance
MedGen UID:
375779
Concept ID:
C1845977
Finding
Source: Orphanet
A mode of inheritance that is observed for recessive traits related to a gene encoded on the X chromosome. In the context of medical genetics, X-linked recessive disorders manifest in males (who have one copy of the X chromosome and are thus hemizygotes), but generally not in female heterozygotes who have one mutant and one normal allele.
 
Related genes: TP53RK, WDR73, OSGEP, TPRKB, LAGE3
 
Monarch Initiative: MONDO:0009627
OMIM®: 251300
OMIM® Phenotypic series: PS251300
Orphanet: ORPHA2065

Definition

Galloway-Mowat syndrome is a rare autosomal recessive neurodegenerative disorder characterized by infantile onset of microcephaly and central nervous system abnormalities resulting in severely delayed psychomotor development. Brain imaging shows cerebellar atrophy and sometimes cerebral atrophy. More variable features include optic atrophy, movement disorders, seizures, and nephrotic syndrome (summary by Vodopiutz et al., 2015). Genetic Heterogeneity of Galloway-Mowat Syndrome See also GAMOS2 (301006), caused by mutation in the LAGE3 gene (300060) on chromosome Xq28; GAMOS3 (617729), caused by mutation in the OSGEP gene (610107) on chromosome 14q11; GAMOS4 (617730), caused by mutation in the TP53RK gene (608679) on chromosome 20q13; GAMOS5 (617731), caused by mutation in the TPRKB gene (608680) on chromosome 2p13; GAMOS6 (618347), caused by mutation in the WDR4 gene (605924) on chromosome 21q22; GAMOS7 (618348), caused by mutation in the NUP107 gene (607617) on chromosome 12q15; GAMOS8 (618349), caused by mutation in the NUP133 gene (607613) on chromosome 1q42; GAMOS9 (619603), caused by mutation in the GON7 gene (617436) on chromosome 14q32; and GAMOS10 (619609), caused by mutation in the YRDC gene (612276) on chromosome 1p34. [from OMIM]

Professional guidelines

PubMed

Braun DA, Rao J, Mollet G, Schapiro D, Daugeron MC, Tan W, Gribouval O, Boyer O, Revy P, Jobst-Schwan T, Schmidt JM, Lawson JA, Schanze D, Ashraf S, Ullmann JFP, Hoogstraten CA, Boddaert N, Collinet B, Martin G, Liger D, Lovric S, Furlano M, Guerrera IC, Sanchez-Ferras O, Hu JF, Boschat AC, Sanquer S, Menten B, Vergult S, De Rocker N, Airik M, Hermle T, Shril S, Widmeier E, Gee HY, Choi WI, Sadowski CE, Pabst WL, Warejko JK, Daga A, Basta T, Matejas V, Scharmann K, Kienast SD, Behnam B, Beeson B, Begtrup A, Bruce M, Ch'ng GS, Lin SP, Chang JH, Chen CH, Cho MT, Gaffney PM, Gipson PE, Hsu CH, Kari JA, Ke YY, Kiraly-Borri C, Lai WM, Lemyre E, Littlejohn RO, Masri A, Moghtaderi M, Nakamura K, Ozaltin F, Praet M, Prasad C, Prytula A, Roeder ER, Rump P, Schnur RE, Shiihara T, Sinha MD, Soliman NA, Soulami K, Sweetser DA, Tsai WH, Tsai JD, Topaloglu R, Vester U, Viskochil DH, Vatanavicharn N, Waxler JL, Wierenga KJ, Wolf MTF, Wong SN, Leidel SA, Truglio G, Dedon PC, Poduri A, Mane S, Lifton RP, Bouchard M, Kannu P, Chitayat D, Magen D, Callewaert B, van Tilbeurgh H, Zenker M, Antignac C, Hildebrandt F
Nat Genet 2017 Oct;49(10):1529-1538. Epub 2017 Aug 14 doi: 10.1038/ng.3933. PMID: 28805828Free PMC Article
Licht C, Eifinger F, Gharib M, Offner G, Michalk DV, Querfeld U
Pediatr Nephrol 2000 Oct;14(12):1077-82. doi: 10.1007/s004670000386. PMID: 11045390

Recent clinical studies

Etiology

Chen Y, Yang Y, Yang Y, Rao J, Bai H
BMC Nephrol 2023 Feb 8;24(1):29. doi: 10.1186/s12882-022-03000-5. PMID: 36755238Free PMC Article
Lin PY, Tseng MH, Zenker M, Rao J, Hildebrandt F, Lin SH, Lin CC, Chang JH, Hsu CH, Lee MD, Lin SP, Tsai JD
Orphanet J Rare Dis 2018 Dec 17;13(1):226. doi: 10.1186/s13023-018-0961-9. PMID: 30558655Free PMC Article
Al-Rakan MA, Abothnain MD, Alrifai MT, Alfadhel M
BMC Ophthalmol 2018 Jun 22;18(1):147. doi: 10.1186/s12886-018-0820-4. PMID: 29929488Free PMC Article
Rosti RO, Dikoglu E, Zaki MS, Abdel-Salam G, Makhseed N, Sese JC, Musaev D, Rosti B, Harbert MJ, Jones MC, Vaux KK, Gleeson JG
Am J Med Genet A 2016 Apr;170A(4):992-8. Epub 2016 Jan 5 doi: 10.1002/ajmg.a.37533. PMID: 27001912Free PMC Article
Vodopiutz J, Seidl R, Prayer D, Khan MI, Mayr JA, Streubel B, Steiß JO, Hahn A, Csaicsich D, Castro C, Assoum M, Müller T, Wieczorek D, Mancini GM, Sadowski CE, Lévy N, Mégarbané A, Godbole K, Schanze D, Hildebrandt F, Delague V, Janecke AR, Zenker M
Hum Mutat 2015 Nov;36(11):1021-8. Epub 2015 Aug 6 doi: 10.1002/humu.22828. PMID: 26123727Free PMC Article

Diagnosis

Chen Y, Yang Y, Yang Y, Rao J, Bai H
BMC Nephrol 2023 Feb 8;24(1):29. doi: 10.1186/s12882-022-03000-5. PMID: 36755238Free PMC Article
Baker E, Weaver D, Massengill S, Mittag D, Juusola J, Demmer L
Pediatr Nephrol 2019 Nov;34(11):2327-2329. Epub 2019 May 8 doi: 10.1007/s00467-019-04261-3. PMID: 31069511
Lin PY, Tseng MH, Zenker M, Rao J, Hildebrandt F, Lin SH, Lin CC, Chang JH, Hsu CH, Lee MD, Lin SP, Tsai JD
Orphanet J Rare Dis 2018 Dec 17;13(1):226. doi: 10.1186/s13023-018-0961-9. PMID: 30558655Free PMC Article
Hou JW, Wang TR
Am J Med Genet 1995 Sep 11;58(3):245-8. doi: 10.1002/ajmg.1320580310. PMID: 8533826
Cooperstone BG, Friedman A, Kaplan BS
Am J Med Genet 1993 Aug 15;47(2):250-4. doi: 10.1002/ajmg.1320470221. PMID: 8213914

Therapy

Fujita A, Tsukaguchi H, Koshimizu E, Nakazato H, Itoh K, Kuraoka S, Komohara Y, Shiina M, Nakamura S, Kitajima M, Tsurusaki Y, Miyatake S, Ogata K, Iijima K, Matsumoto N, Miyake N
Ann Neurol 2018 Dec;84(6):814-828. doi: 10.1002/ana.25370. PMID: 30427554
Naidu GD, Deepthi P, RajaKarthik K, Sriram S, Swarnalatha G, Gangadhar T
Saudi J Kidney Dis Transpl 2017 Sep-Oct;28(5):1188-1191. doi: 10.4103/1319-2442.215154. PMID: 28937085
Licht C, Eifinger F, Gharib M, Offner G, Michalk DV, Querfeld U
Pediatr Nephrol 2000 Oct;14(12):1077-82. doi: 10.1007/s004670000386. PMID: 11045390

Prognosis

Mann N, Mzoughi S, Schneider R, Kühl SJ, Schanze D, Klämbt V, Lovric S, Mao Y, Shi S, Tan W, Kühl M, Onuchic-Whitford AC, Treimer E, Kitzler TM, Kause F, Schumann S, Nakayama M, Buerger F, Shril S, van der Ven AT, Majmundar AJ, Holton KM, Kolb A, Braun DA, Rao J, Jobst-Schwan T, Mildenberger E, Lennert T, Kuechler A, Wieczorek D, Gross O, Ermisch-Omran B, Werberger A, Skalej M, Janecke AR, Soliman NA, Mane SM, Lifton RP, Kadlec J, Guccione E, Schmeisser MJ, Zenker M, Hildebrandt F
J Am Soc Nephrol 2021 Mar;32(3):580-596. Epub 2021 Feb 16 doi: 10.1681/ASN.2020040490. PMID: 33593823Free PMC Article
Baker E, Weaver D, Massengill S, Mittag D, Juusola J, Demmer L
Pediatr Nephrol 2019 Nov;34(11):2327-2329. Epub 2019 May 8 doi: 10.1007/s00467-019-04261-3. PMID: 31069511
Al-Rakan MA, Abothnain MD, Alrifai MT, Alfadhel M
BMC Ophthalmol 2018 Jun 22;18(1):147. doi: 10.1186/s12886-018-0820-4. PMID: 29929488Free PMC Article
Horton AL, Smith JK, Strauss RA
Prenat Diagn 2009 Mar;29(3):280-2. doi: 10.1002/pd.2216. PMID: 19177459
Cooperstone BG, Friedman A, Kaplan BS
Am J Med Genet 1993 Aug 15;47(2):250-4. doi: 10.1002/ajmg.1320470221. PMID: 8213914

Clinical prediction guides

Alves GR, Javaroni JB, Moura APGE, Consolaro A, Segato RAB
Spec Care Dentist 2024 Jul-Aug;44(4):1054-1058. Epub 2024 Feb 6 doi: 10.1111/scd.12971. PMID: 38321585
Treimer E, Niedermayer K, Schumann S, Zenker M, Schmeisser MJ, Kühl SJ
Gene Expr Patterns 2021 Dec;42:119215. Epub 2021 Oct 4 doi: 10.1016/j.gep.2021.119215. PMID: 34619372
Braun DA, Shril S, Sinha A, Schneider R, Tan W, Ashraf S, Hermle T, Jobst-Schwan T, Widmeier E, Majmundar AJ, Daga A, Warejko JK, Nakayama M, Schapiro D, Chen J, Airik M, Rao J, Schmidt JM, Hoogstraten CA, Hugo H, Meena J, Lek M, Laricchia KM, Bagga A, Hildebrandt F
Am J Med Genet A 2018 Nov;176(11):2460-2465. Epub 2018 Aug 6 doi: 10.1002/ajmg.a.40489. PMID: 30079490Free PMC Article
Rosti RO, Dikoglu E, Zaki MS, Abdel-Salam G, Makhseed N, Sese JC, Musaev D, Rosti B, Harbert MJ, Jones MC, Vaux KK, Gleeson JG
Am J Med Genet A 2016 Apr;170A(4):992-8. Epub 2016 Jan 5 doi: 10.1002/ajmg.a.37533. PMID: 27001912Free PMC Article
Zenker M, Tralau T, Lennert T, Pitz S, Mark K, Madlon H, Dötsch J, Reis A, Müntefering H, Neumann LM
Am J Med Genet A 2004 Oct 1;130A(2):138-45. doi: 10.1002/ajmg.a.30310. PMID: 15372515

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