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Frontometaphyseal dysplasia(FMD1)

MedGen UID:
82703
Concept ID:
C0265293
Congenital Abnormality
Synonym: FMD1
SNOMED CT: Frontometaphyseal dysplasia (62803002); FMD - Frontometaphyseal dysplasia (62803002)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
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 heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
X-linked dominant inheritance
MedGen UID:
376232
Concept ID:
C1847879
Finding
Source: Orphanet
A mode of inheritance that is observed for dominant traits related to a gene encoded on the X chromosome. In the context of medical genetics, X-linked dominant disorders tend to manifest very severely in affected males. The severity of manifestation in females may depend on the degree of skewed X inactivation.
 
Related genes: MAP3K7, FLNA
 
Monarch Initiative: MONDO:0015942
OMIM® Phenotypic series: PS305620
Orphanet: ORPHA1826

Definition

Frontometaphyseal dysplasia-1 (FMD1) is 1 of 4 otopalatodigital syndromes caused by mutations in the FLNA gene. The disorders, which include otopalatodigital syndrome-1 (OPD1; 311300), otopalatodigital syndrome-2 (OPD2; 304120), and Melnick-Needles syndrome (MNS; 309350), constitute a phenotypic spectrum. At the mild end of the spectrum, males with OPD1 have cleft palate and mild skeletal anomalies with conductive deafness caused by ossicular anomalies. FMD1 is characterized by a generalized skeletal dysplasia, deafness, and urogenital defects. Males with OPD2 have disabling skeletal anomalies in addition to variable malformations in the hindbrain, heart, intestines, and kidneys that frequently lead to perinatal death. The most severe phenotype, MNS, is characterized by a skeletal dysplasia in the heterozygote. Affected males exhibit severe malformations similar to those observed in individuals with OPD2, resulting in prenatal lethality or death in the first few months of life (review by Robertson, 2005). Verloes et al. (2000) suggested that these disorders constitute a single entity, which they termed 'frontootopalatodigital osteodysplasia.' Genetic Heterogeneity of Frontometaphyseal Dysplasia Frontometaphyseal dysplasia-2 (FMD2; 617137) is caused by mutation in the MAP3K7 gene (602614) on chromosome 6q15. [from OMIM]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
Follow this link to review classifications for Frontometaphyseal dysplasia in Orphanet.

Professional guidelines

PubMed

van Woerden GM, Senden R, de Konink C, Trezza RA, Baban A, Bassetti JA, van Bever Y, Bird LM, van Bon BW, Brooks AS, Guan Q, Klee EW, Marcelis C, Rosado JM, Schimmenti LA, Shikany AR, Terhal PA, Nicole Weaver K, Wessels MW, van Wieringen H, Hurst AC, Gooch CF, Steindl K, Joset P, Rauch A, Tartaglia M, Niceta M, Elgersma Y, Demirdas S
Hum Mutat 2022 Oct;43(10):1377-1395. Epub 2022 Jul 29 doi: 10.1002/humu.24425. PMID: 35730652Free PMC Article
Bertola D, Passos-Bueno MR, Pereira A, Kim C, Morgan T, Robertson SP
Am J Med Genet A 2015 May;167A(5):1161-4. Epub 2015 Mar 28 doi: 10.1002/ajmg.a.36981. PMID: 25820619

Recent clinical studies

Etiology

van Woerden GM, Senden R, de Konink C, Trezza RA, Baban A, Bassetti JA, van Bever Y, Bird LM, van Bon BW, Brooks AS, Guan Q, Klee EW, Marcelis C, Rosado JM, Schimmenti LA, Shikany AR, Terhal PA, Nicole Weaver K, Wessels MW, van Wieringen H, Hurst AC, Gooch CF, Steindl K, Joset P, Rauch A, Tartaglia M, Niceta M, Elgersma Y, Demirdas S
Hum Mutat 2022 Oct;43(10):1377-1395. Epub 2022 Jul 29 doi: 10.1002/humu.24425. PMID: 35730652Free PMC Article
Gangadaran P, Chaudhry C, Panigrahi I, Kumari A, Kaur A
Am J Med Genet A 2021 May;185(5):1550-1553. Epub 2021 Feb 22 doi: 10.1002/ajmg.a.62134. PMID: 33615695
Basart H, van de Kar A, Adès L, Cho TJ, Carter E, Maas SM, Wilson LC, van der Horst CM, Wade EM, Robertson SP, Hennekam RC
Am J Med Genet A 2015 Jun;167(6):1215-22. Epub 2015 Apr 21 doi: 10.1002/ajmg.a.37044. PMID: 25899317
Faden MA, Krakow D, Ezgu F, Rimoin DL, Lachman RS
Am J Med Genet A 2009 Jun;149A(6):1334-45. doi: 10.1002/ajmg.a.32253. PMID: 19444897Free PMC Article
Robertson SP, Jenkins ZA, Morgan T, Adès L, Aftimos S, Boute O, Fiskerstrand T, Garcia-Miñaur S, Grix A, Green A, Der Kaloustian V, Lewkonia R, McInnes B, van Haelst MM, Mancini G, Illés T, Mortier G, Newbury-Ecob R, Nicholson L, Scott CI, Ochman K, Brozek I, Shears DJ, Superti-Furga A, Suri M, Whiteford M, Wilkie AO, Krakow D
Am J Med Genet A 2006 Aug 15;140(16):1726-36. doi: 10.1002/ajmg.a.31322. PMID: 16835913

Diagnosis

Sun Z, Xu Z, Sun J, Liu J, Ma H
Arch Argent Pediatr 2022 Dec;120(6):e278-e282. Epub 2022 Nov 1 doi: 10.5546/aap.2022.eng.e278. PMID: 36374066
van Woerden GM, Senden R, de Konink C, Trezza RA, Baban A, Bassetti JA, van Bever Y, Bird LM, van Bon BW, Brooks AS, Guan Q, Klee EW, Marcelis C, Rosado JM, Schimmenti LA, Shikany AR, Terhal PA, Nicole Weaver K, Wessels MW, van Wieringen H, Hurst AC, Gooch CF, Steindl K, Joset P, Rauch A, Tartaglia M, Niceta M, Elgersma Y, Demirdas S
Hum Mutat 2022 Oct;43(10):1377-1395. Epub 2022 Jul 29 doi: 10.1002/humu.24425. PMID: 35730652Free PMC Article
Yapijakis C, Vylliotis A, Angelopoulou A, Adamopoulou M, Chrousos GP, Voumvourakis C
Adv Exp Med Biol 2021;1339:319-323. doi: 10.1007/978-3-030-78787-5_38. PMID: 35023120
Naudion S, Moutton S, Coupry I, Sole G, Deforges J, Guerineau E, Hubert C, Deves S, Pilliod J, Rooryck C, Abel C, Le Breton F, Collardeau-Frachon S, Cordier MP, Delezoide AL, Goldenberg A, Loget P, Melki J, Odent S, Patrier S, Verloes A, Viot G, Blesson S, Bessières B, Lacombe D, Arveiler B, Goizet C, Fergelot P
Clin Genet 2016 Mar;89(3):371-7. Epub 2015 Oct 29 doi: 10.1111/cge.12679. PMID: 26404489
Robertson SP, Jenkins ZA, Morgan T, Adès L, Aftimos S, Boute O, Fiskerstrand T, Garcia-Miñaur S, Grix A, Green A, Der Kaloustian V, Lewkonia R, McInnes B, van Haelst MM, Mancini G, Illés T, Mortier G, Newbury-Ecob R, Nicholson L, Scott CI, Ochman K, Brozek I, Shears DJ, Superti-Furga A, Suri M, Whiteford M, Wilkie AO, Krakow D
Am J Med Genet A 2006 Aug 15;140(16):1726-36. doi: 10.1002/ajmg.a.31322. PMID: 16835913

Prognosis

Wade EM, Jenkins ZA, Morgan T, Gimenez G, Gibson H, Peng H, Sanchez Russo R, Skraban CM, Bedoukian E, Robertson SP
Am J Med Genet A 2021 Dec;185(12):3675-3682. Epub 2021 Jul 17 doi: 10.1002/ajmg.a.62424. PMID: 34272929
Thieu T, Milman T, Bhatti TR, Eagle RC Jr
J Pediatr Ophthalmol Strabismus 2020 Jan 24;57:e8-e11. doi: 10.3928/01913913-20191230-02. PMID: 31978233
Xie H, Xue L, Hua W, Jia B, Zhang L, Li L
Medicine (Baltimore) 2018 Jul;97(28):e11283. doi: 10.1097/MD.0000000000011283. PMID: 29995760Free PMC Article
Santos HH, Garcia PP, Pereira L, Leão LL, Aguiar RA, Lana AM, Carvalho MR, Aguiar MJ
Am J Med Genet A 2010 Mar;152A(3):726-31. doi: 10.1002/ajmg.a.33260. PMID: 20186808
Zenker M, Nährlich L, Sticht H, Reis A, Horn D
Am J Med Genet A 2006 May 15;140(10):1069-73. doi: 10.1002/ajmg.a.31213. PMID: 16596676

Clinical prediction guides

Wade EM, Jenkins ZA, Morgan T, Gimenez G, Gibson H, Peng H, Sanchez Russo R, Skraban CM, Bedoukian E, Robertson SP
Am J Med Genet A 2021 Dec;185(12):3675-3682. Epub 2021 Jul 17 doi: 10.1002/ajmg.a.62424. PMID: 34272929
Dissanayake R, Senanayake MP, Fernando J, Robertson SP, Dissanayake VHW, Sirisena ND
Am J Med Genet A 2021 Apr;185(4):1317-1320. Epub 2020 Dec 29 doi: 10.1002/ajmg.a.62058. PMID: 33372358
Costantini A, Wallgren-Pettersson C, Mäkitie O
Eur J Med Genet 2018 Oct;61(10):612-615. Epub 2018 Apr 14 doi: 10.1016/j.ejmg.2018.04.004. PMID: 29660408
Robertson SP, Jenkins ZA, Morgan T, Adès L, Aftimos S, Boute O, Fiskerstrand T, Garcia-Miñaur S, Grix A, Green A, Der Kaloustian V, Lewkonia R, McInnes B, van Haelst MM, Mancini G, Illés T, Mortier G, Newbury-Ecob R, Nicholson L, Scott CI, Ochman K, Brozek I, Shears DJ, Superti-Furga A, Suri M, Whiteford M, Wilkie AO, Krakow D
Am J Med Genet A 2006 Aug 15;140(16):1726-36. doi: 10.1002/ajmg.a.31322. PMID: 16835913
Holthusen W, Holt JF, Stoeckenius M
Pediatr Radiol 1975 Jun 13;3(3):137-44. doi: 10.1007/BF01006898. PMID: 1233427

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