U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Links from Gene

Neuromuscular disorder, congenital, with dysmorphic facies

MedGen UID:
1053186
Concept ID:
CN377270
Disease or Syndrome
Synonym: neuromuscular disorder, congenital, with dysmorphic facies
 
Gene (location): FILIP1 (6q14.1)
 
Monarch Initiative: MONDO:0958332
OMIM®: 620775

Definition

Congenital neuromuscular disorder with dysmorphic facies (NMDF) is an autosomal recessive disorder characterized by impaired skeletal muscle development, usually resulting in hypotonia and secondary joint contractures, and dysmorphic facial features. Features are apparent from birth. Affected individuals may show motor delay, speech delay, and impaired intellectual development. The severity of the disorder is highly variable (Schnabel et al., 2023; Roos et al., 2023). [from OMIM]

Professional guidelines

PubMed

Mroczek M, Iyadurai S
Int J Mol Sci 2023 Jan 23;24(3) doi: 10.3390/ijms24032260. PMID: 36768582Free PMC Article
de la Fuente Díez Y, Olvera Morales O, Chen López CY, Tovilla Canales JL, Nava Castañeda A
Arch Soc Esp Oftalmol (Engl Ed) 2020 Jan;95(1):9-14. Epub 2019 Dec 2 doi: 10.1016/j.oftal.2019.09.008. PMID: 31806264
Anderson PJ, Barker JH, David DJ
World J Orthod 2005 Summer;6(2):156-60. PMID: 15952552

Recent clinical studies

Etiology

Landau Prat D, Zhao CS, Ramakrishnan M, Revere KE, Katowitz WR, Katowitz JA
Can J Ophthalmol 2024 Aug;59(4):264-269. Epub 2023 Jun 24 doi: 10.1016/j.jcjo.2023.06.002. PMID: 37364855
Garren B, Stephan M, Hogue JS
Am J Med Genet A 2020 Jan;182(1):195-200. Epub 2019 Nov 7 doi: 10.1002/ajmg.a.61395. PMID: 31697451
Cao H, Alrejaye N, Klein OD, Goodwin AF, Oberoi S
Orthod Craniofac Res 2017 Jun;20 Suppl 1(Suppl 1):32-38. doi: 10.1111/ocr.12144. PMID: 28643916Free PMC Article
Stevenson DA, Allen S, Tidyman WE, Carey JC, Viskochil DH, Stevens A, Hanson H, Sheng X, Thompson BA, Okumura MJ, Reinker K, Johnson B, Rauen KA
Muscle Nerve 2012 Sep;46(3):394-9. doi: 10.1002/mus.23324. PMID: 22907230
Narayanan V
Br J Oral Maxillofac Surg 1996 Oct;34(5):446-9. doi: 10.1016/s0266-4356(96)90105-9. PMID: 8909740

Diagnosis

Alzahem TA, AlTheeb A, Ba-Abbad R
Ophthalmic Genet 2024 Aug;45(4):404-408. Epub 2024 Apr 15 doi: 10.1080/13816810.2024.2321871. PMID: 38619019
Normatov I, Sentongo T
Pediatr Ann 2019 Nov 1;48(11):e441-e447. doi: 10.3928/19382359-20191018-01. PMID: 31710363
Laughlin RS, Niu Z, Wieben E, Milone M
Mol Genet Genomic Med 2017 Nov;5(6):800-804. Epub 2017 Oct 4 doi: 10.1002/mgg3.338. PMID: 29178655Free PMC Article
Cao H, Alrejaye N, Klein OD, Goodwin AF, Oberoi S
Orthod Craniofac Res 2017 Jun;20 Suppl 1(Suppl 1):32-38. doi: 10.1111/ocr.12144. PMID: 28643916Free PMC Article
Tartaglia M, Gelb BD, Zenker M
Best Pract Res Clin Endocrinol Metab 2011 Feb;25(1):161-79. doi: 10.1016/j.beem.2010.09.002. PMID: 21396583Free PMC Article

Therapy

de la Fuente Díez Y, Olvera Morales O, Chen López CY, Tovilla Canales JL, Nava Castañeda A
Arch Soc Esp Oftalmol (Engl Ed) 2020 Jan;95(1):9-14. Epub 2019 Dec 2 doi: 10.1016/j.oftal.2019.09.008. PMID: 31806264
Spitzer AB, Shaw KA, Schmitz M, Devito DP, Murphy JS
Spine Deform 2019 Jan;7(1):158-162. doi: 10.1016/j.jspd.2018.05.002. PMID: 30587310
Rauen KA, Banerjee A, Bishop WR, Lauchle JO, McCormick F, McMahon M, Melese T, Munster PN, Nadaf S, Packer RJ, Sebolt-Leopold J, Viskochil DH
Am J Med Genet C Semin Med Genet 2011 May 15;157C(2):136-46. Epub 2011 Apr 14 doi: 10.1002/ajmg.c.30294. PMID: 21495172Free PMC Article
Jinbo AK
J Wound Ostomy Continence Nurs 2004 Nov-Dec;31(6):336-50. doi: 10.1097/00152192-200411000-00005. PMID: 15867709
Voit T, Kramer H, Thomas C, Wechsler W, Reichmann H, Lenard HG
Eur J Pediatr 1991 May;150(7):521-6. doi: 10.1007/BF01958438. PMID: 1915507

Prognosis

Landau Prat D, Zhao CS, Ramakrishnan M, Revere KE, Katowitz WR, Katowitz JA
Can J Ophthalmol 2024 Aug;59(4):264-269. Epub 2023 Jun 24 doi: 10.1016/j.jcjo.2023.06.002. PMID: 37364855
de la Fuente Díez Y, Olvera Morales O, Chen López CY, Tovilla Canales JL, Nava Castañeda A
Arch Soc Esp Oftalmol (Engl Ed) 2020 Jan;95(1):9-14. Epub 2019 Dec 2 doi: 10.1016/j.oftal.2019.09.008. PMID: 31806264
Rodríguez Cruz PM, Cossins J, Estephan EP, Munell F, Selby K, Hirano M, Maroofin R, Mehrjardi MYV, Chow G, Carr A, Manzur A, Robb S, Munot P, Wei Liu W, Banka S, Fraser H, De Goede C, Zanoteli E, Conti Reed U, Sage A, Gratacos M, Macaya A, Dusl M, Senderek J, Töpf A, Hofer M, Knight R, Ramdas S, Jayawant S, Lochmüller H, Palace J, Beeson D
Brain 2019 Jun 1;142(6):1547-1560. doi: 10.1093/brain/awz107. PMID: 31081514Free PMC Article
Yamaguchi T, Takano K, Inaba Y, Morikawa M, Motobayashi M, Kawamura R, Wakui K, Nishi E, Hirabayashi SI, Fukushima Y, Kato H, Takahashi J, Kosho T
Am J Med Genet A 2019 Jun;179(6):948-957. Epub 2019 Apr 2 doi: 10.1002/ajmg.a.61142. PMID: 30941898
Folkmann AW, Dawson TR, Wente SR
Adv Biol Regul 2014 Jan;54:74-91. Epub 2013 Nov 13 doi: 10.1016/j.jbior.2013.10.002. PMID: 24275432Free PMC Article

Clinical prediction guides

Harel T, Spicher C, Scheer E, Buchan JG, Cech J, Folland C, Frey T, Holtz AM, Innes AM, Keren B, Macken WL, Marcelis C, Otten CE, Paolucci SA, Petit F, Pfundt R, Pitceathly RDS, Rauch A, Ravenscroft G, Sanchev R, Steindl K, Tammer F, Tyndall A, Devys D, Vincent SD, Elpeleg O, Tora L
Brain 2024 Aug 1;147(8):2732-2744. doi: 10.1093/brain/awae160. PMID: 38753057
Natera-de Benito D, Jurgens JA, Yeung A, Zaharieva IT, Manzur A, DiTroia SP, Di Gioia SA, Pais L, Pini V, Barry BJ, Chan WM, Elder JE, Christodoulou J, Hay E, England EM, Munot P, Hunter DG, Feng L, Ledoux D, O'Donnell-Luria A, Phadke R, Engle EC, Sarkozy A, Muntoni F
Hum Mutat 2022 Apr;43(4):487-498. Epub 2022 Feb 3 doi: 10.1002/humu.24333. PMID: 35077597Free PMC Article
Bizaoui V, Gage J, Brar R, Rauen KA, Weiss LA
Am J Med Genet B Neuropsychiatr Genet 2018 Jun;177(4):434-446. Epub 2018 Apr 16 doi: 10.1002/ajmg.b.32632. PMID: 29659143Free PMC Article
Uzak AS, Fryns JP, Dundar M
Genet Couns 2014;25(2):159-69. PMID: 25059014
Folkmann AW, Dawson TR, Wente SR
Adv Biol Regul 2014 Jan;54:74-91. Epub 2013 Nov 13 doi: 10.1016/j.jbior.2013.10.002. PMID: 24275432Free PMC Article

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...