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Distal myopathy with anterior tibial onset(DMAT)

MedGen UID:
335706
Concept ID:
C1847532
Disease or Syndrome
Synonym: Myopathy, Distal, with Anterior Tibial Onset
SNOMED CT: Distal myopathy with anterior tibial onset (782675008); Distal anterior compartment myopathy (782675008)
 
Gene (location): DYSF (2p13.2)
 
Monarch Initiative: MONDO:0011721
OMIM®: 606768
Orphanet: ORPHA178400

Definition

A rare genetic neuromuscular disease with characteristics of a progressive muscle weakness starting in the anterior tibial muscles, later involving lower and upper limb muscles, associated with an increased serum creatine kinase levels and absence of dysferlin on muscle biopsy. There is evidence the disease is caused by homozygous mutation in the gene encoding dysferlin (DYSF) on chromosome 2p13. Patients become wheelchair dependent. [from SNOMEDCT_US]

Clinical features

From HPO
Loss of ambulation
MedGen UID:
332305
Concept ID:
C1836843
Finding
Inability to walk in a person who previous had the ability to walk.
Myopathy
MedGen UID:
10135
Concept ID:
C0026848
Disease or Syndrome
A disorder of muscle unrelated to impairment of innervation or neuromuscular junction.
Proximal muscle weakness
MedGen UID:
113169
Concept ID:
C0221629
Finding
A lack of strength of the proximal muscles.
Elevated circulating creatine kinase concentration
MedGen UID:
69128
Concept ID:
C0241005
Finding
An elevation of the level of the enzyme creatine kinase (also known as creatine phosphokinase (CK; EC 2.7.3.2) in the blood. CK levels can be elevated in a number of clinical disorders such as myocardial infarction, rhabdomyolysis, and muscular dystrophy.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVDistal myopathy with anterior tibial onset
Follow this link to review classifications for Distal myopathy with anterior tibial onset in Orphanet.

Recent clinical studies

Etiology

Belhassen I, Laroussi S, Sakka S, Rekik S, Lahkim L, Dammak M, Authier FJ, Mhiri C
Neuromuscul Disord 2023 Oct;33(10):718-727. Epub 2023 Aug 18 doi: 10.1016/j.nmd.2023.08.007. PMID: 37716854
Anwar S, Yokota T
Methods Mol Biol 2023;2587:183-196. doi: 10.1007/978-1-0716-2772-3_11. PMID: 36401031
Katz J, Labilloy A, Lee A
BMC Pediatr 2022 Aug 30;22(1):515. doi: 10.1186/s12887-022-03561-2. PMID: 36042458Free PMC Article
Ivanova A, Smirnikhina S, Lavrov A
Clin Genet 2022 Dec;102(6):465-473. Epub 2022 Sep 6 doi: 10.1111/cge.14216. PMID: 36029111
Rekik S, Sakka S, Romdhane SB, Amer YB, Lehkim L, Farhat N, Mahfoudh KB, Authier FJ, Dammak M, Mhiri C
Mol Biol Rep 2020 Aug;47(8):5755-5761. Epub 2020 Jul 14 doi: 10.1007/s11033-020-05643-9. PMID: 32666437

Diagnosis

Park J, Moon YJ, Kim DS
Genes (Basel) 2023 Jan 12;14(1) doi: 10.3390/genes14010200. PMID: 36672942Free PMC Article
Urtizberea JA, Bassez G, Leturcq F, Nguyen K, Krahn M, Levy N
Neurol India 2008 Jul-Sep;56(3):289-97. doi: 10.4103/0028-3886.43447. PMID: 18974555

Therapy

Maruyama R, Yokota T
Methods Mol Biol 2018;1828:79-90. doi: 10.1007/978-1-4939-8651-4_5. PMID: 30171536
Touznik A, Lee JJ, Yokota T
Expert Opin Biol Ther 2014 Jun;14(6):809-19. Epub 2014 Mar 12 doi: 10.1517/14712598.2014.896335. PMID: 24620745
Aartsma-Rus A, Singh KH, Fokkema IF, Ginjaar IB, van Ommen GJ, den Dunnen JT, van der Maarel SM
Eur J Hum Genet 2010 Aug;18(8):889-94. Epub 2010 Feb 10 doi: 10.1038/ejhg.2010.4. PMID: 20145676Free PMC Article

Prognosis

Belhassen I, Laroussi S, Sakka S, Rekik S, Lahkim L, Dammak M, Authier FJ, Mhiri C
Neuromuscul Disord 2023 Oct;33(10):718-727. Epub 2023 Aug 18 doi: 10.1016/j.nmd.2023.08.007. PMID: 37716854
Liu J, Aoki M, Illa I, Wu C, Fardeau M, Angelini C, Serrano C, Urtizberea JA, Hentati F, Hamida MB, Bohlega S, Culper EJ, Amato AA, Bossie K, Oeltjen J, Bejaoui K, McKenna-Yasek D, Hosler BA, Schurr E, Arahata K, de Jong PJ, Brown RH Jr
Nat Genet 1998 Sep;20(1):31-6. doi: 10.1038/1682. PMID: 9731526

Clinical prediction guides

Belhassen I, Laroussi S, Sakka S, Rekik S, Lahkim L, Dammak M, Authier FJ, Mhiri C
Neuromuscul Disord 2023 Oct;33(10):718-727. Epub 2023 Aug 18 doi: 10.1016/j.nmd.2023.08.007. PMID: 37716854
Liu J, Aoki M, Illa I, Wu C, Fardeau M, Angelini C, Serrano C, Urtizberea JA, Hentati F, Hamida MB, Bohlega S, Culper EJ, Amato AA, Bossie K, Oeltjen J, Bejaoui K, McKenna-Yasek D, Hosler BA, Schurr E, Arahata K, de Jong PJ, Brown RH Jr
Nat Genet 1998 Sep;20(1):31-6. doi: 10.1038/1682. PMID: 9731526

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