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Centronuclear myopathy(CNM)

MedGen UID:
104495
Concept ID:
C0175709
Disease or Syndrome
Synonyms: Centronuclear myopathy, congenital; CNM; Myotubular myopathy
SNOMED CT: Myotubular myopathy (82077006); Centronuclear myopathy (82077006)
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).
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 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: MTMR14, MTM1, DNM2, BIN1
 
Monarch Initiative: MONDO:0018947
OMIM® Phenotypic series: PS160150
Orphanet: ORPHA595

Definition

Centronuclear myopathy is a condition characterized by muscle weakness (myopathy) and wasting (atrophy) in the skeletal muscles, which are the muscles used for movement. The severity of centronuclear myopathy varies among affected individuals, even among members of the same family.

People with centronuclear myopathy begin experiencing muscle weakness at any time from birth to early adulthood. The muscle weakness slowly worsens over time and can lead to delayed development of motor skills, such as crawling or walking; muscle pain during exercise; and difficulty walking. Some affected individuals may need wheelchair assistance as the muscles atrophy and weakness becomes more severe. In rare instances, the muscle weakness improves over time.

A key feature of centronuclear myopathy is the displacement of the nucleus in muscle cells, which can be viewed under a microscope. Normally the nucleus is found at the edges of the rod-shaped muscle cells, but in people with centronuclear myopathy the nucleus is located in the center of these cells. How the change in location of the nucleus affects muscle cell function is unknown.

Some people with centronuclear myopathy experience mild to severe breathing problems related to the weakness of muscles needed for breathing. People with centronuclear myopathy may have droopy eyelids (ptosis) and weakness in other facial muscles, including the muscles that control eye movement. People with this condition may also have foot abnormalities, a high arch in the roof of the mouth (high-arched palate), and abnormal side-to-side curvature of the spine (scoliosis). Rarely, individuals with centronuclear myopathy have a weakened heart muscle (cardiomyopathy), disturbances in nerve function (neuropathy), or intellectual disability. [from MedlinePlus Genetics]

Professional guidelines

PubMed

Kušíková K, Šoltýsová A, Ficek A, Feichtinger RG, Mayr JA, Škopková M, Gašperíková D, Kolníková M, Ornig K, Kalev O, Weis S, Weis D
Genes (Basel) 2023 Dec 3;14(12) doi: 10.3390/genes14122174. PMID: 38136996Free PMC Article
Gómez-Oca R, Cowling BS, Laporte J
Int J Mol Sci 2021 Oct 21;22(21) doi: 10.3390/ijms222111377. PMID: 34768808Free PMC Article
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J Neurol 2015 Jul;262(7):1728-40. Epub 2015 May 10 doi: 10.1007/s00415-015-7757-9. PMID: 25957634

Recent clinical studies

Etiology

Schartner V, Romero NB, Donkervoort S, Treves S, Munot P, Pierson TM, Dabaj I, Malfatti E, Zaharieva IT, Zorzato F, Abath Neto O, Brochier G, Lornage X, Eymard B, Taratuto AL, Böhm J, Gonorazky H, Ramos-Platt L, Feng L, Phadke R, Bharucha-Goebel DX, Sumner CJ, Bui MT, Lacene E, Beuvin M, Labasse C, Dondaine N, Schneider R, Thompson J, Boland A, Deleuze JF, Matthews E, Pakleza AN, Sewry CA, Biancalana V, Quijano-Roy S, Muntoni F, Fardeau M, Bönnemann CG, Laporte J
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Neurology 2013 Oct 1;81(14):1205-14. Epub 2013 Aug 23 doi: 10.1212/WNL.0b013e3182a6ca62. PMID: 23975875Free PMC Article
Engel AG, Shen XM, Selcen D, Sine S
Ann N Y Acad Sci 2012 Dec;1275(1):54-62. doi: 10.1111/j.1749-6632.2012.06803.x. PMID: 23278578Free PMC Article
Riggs JE, Bodensteiner JB, Schochet SS Jr
Neurol Clin 2003 Nov;21(4):779-94; v-vi. doi: 10.1016/s0733-8619(03)00013-6. PMID: 14743649
Bornemann A, Goebel HH
Brain Pathol 2001 Apr;11(2):206-17. doi: 10.1111/j.1750-3639.2001.tb00393.x. PMID: 11303796Free PMC Article

Diagnosis

Bayonés L, Guerra-Fernández MJ, Hinostroza F, Báez-Matus X, Vásquez-Navarrete J, Gallo LI, Parra S, Martínez AD, González-Jamett A, Marengo FD, Cárdenas AM
Int J Mol Sci 2022 Sep 8;23(18) doi: 10.3390/ijms231810363. PMID: 36142275Free PMC Article
Lawal TA, Todd JJ, Meilleur KG
Neurotherapeutics 2018 Oct;15(4):885-899. doi: 10.1007/s13311-018-00677-1. PMID: 30406384Free PMC Article
Ceyhan-Birsoy O, Agrawal PB, Hidalgo C, Schmitz-Abe K, DeChene ET, Swanson LC, Soemedi R, Vasli N, Iannaccone ST, Shieh PB, Shur N, Dennison JM, Lawlor MW, Laporte J, Markianos K, Fairbrother WG, Granzier H, Beggs AH
Neurology 2013 Oct 1;81(14):1205-14. Epub 2013 Aug 23 doi: 10.1212/WNL.0b013e3182a6ca62. PMID: 23975875Free PMC Article
Engel AG, Shen XM, Selcen D, Sine S
Ann N Y Acad Sci 2012 Dec;1275(1):54-62. doi: 10.1111/j.1749-6632.2012.06803.x. PMID: 23278578Free PMC Article
Bornemann A, Goebel HH
Brain Pathol 2001 Apr;11(2):206-17. doi: 10.1111/j.1750-3639.2001.tb00393.x. PMID: 11303796Free PMC Article

Therapy

Giraud Q, Spiegelhalter C, Messaddeq N, Laporte J
Brain 2023 Oct 3;146(10):4158-4173. doi: 10.1093/brain/awad251. PMID: 37490306Free PMC Article
Fouarge E, Monseur A, Boulanger B, Annoussamy M, Seferian AM, De Lucia S, Lilien C, Thielemans L, Paradis K, Cowling BS, Freitag C, Carlin BP, Servais L; NatHis-MTM Study Group
Orphanet J Rare Dis 2021 Jan 6;16(1):3. doi: 10.1186/s13023-020-01663-7. PMID: 33407688Free PMC Article
Jungbluth H, Muntoni F
Semin Pediatr Neurol 2019 Apr;29:71-82. Epub 2019 Jan 16 doi: 10.1016/j.spen.2019.01.004. PMID: 31060727
Smith BK, Renno MS, Green MM, Sexton TM, Lawson LA, Martin AD, Corti M, Byrne BJ
Muscle Nerve 2016 Feb;53(2):214-21. Epub 2015 Dec 29 doi: 10.1002/mus.24899. PMID: 26351754Free PMC Article
Mrak RE
Muscle Nerve 1982 Oct;5(8):637-9. doi: 10.1002/mus.880050808. PMID: 7155175

Prognosis

Fujise K, Okubo M, Abe T, Yamada H, Takei K, Nishino I, Takeda T, Noguchi S
Hum Mutat 2022 Feb;43(2):169-179. Epub 2021 Dec 19 doi: 10.1002/humu.24307. PMID: 34837441
Deneubourg C, Ramm M, Smith LJ, Baron O, Singh K, Byrne SC, Duchen MR, Gautel M, Eskelinen EL, Fanto M, Jungbluth H
Autophagy 2022 Mar;18(3):496-517. Epub 2021 Aug 19 doi: 10.1080/15548627.2021.1943177. PMID: 34130600Free PMC Article
Fouarge E, Monseur A, Boulanger B, Annoussamy M, Seferian AM, De Lucia S, Lilien C, Thielemans L, Paradis K, Cowling BS, Freitag C, Carlin BP, Servais L; NatHis-MTM Study Group
Orphanet J Rare Dis 2021 Jan 6;16(1):3. doi: 10.1186/s13023-020-01663-7. PMID: 33407688Free PMC Article
Srinivasan S, Dharmarajan V, Reed DK, Griffin PR, Schmid SL
EMBO J 2016 Feb 15;35(4):443-57. Epub 2016 Jan 18 doi: 10.15252/embj.201593477. PMID: 26783363Free PMC Article
Fan HC, Lee CM, Harn HJ, Cheng SN, Yuh YS
Am J Perinatol 2003 May;20(4):173-9. doi: 10.1055/s-2003-40603. PMID: 12874727

Clinical prediction guides

Zambo B, Edelweiss E, Morlet B, Negroni L, Pajkos M, Dosztanyi Z, Ostergaard S, Trave G, Laporte J, Gogl G
Elife 2024 Jul 12;13 doi: 10.7554/eLife.95397. PMID: 38995680Free PMC Article
Samsó P, Koch PA, Posor Y, Lo WT, Belabed H, Nazare M, Laporte J, Haucke V
Proc Natl Acad Sci U S A 2022 Oct 4;119(40):e2202236119. Epub 2022 Sep 26 doi: 10.1073/pnas.2202236119. PMID: 36161941Free PMC Article
Wang X, Jia Y, Zhao J, Lesner NP, Menezes CJ, Shelton SD, Venigalla SSK, Xu J, Cai C, Mishra P
J Clin Invest 2022 Dec 1;132(23) doi: 10.1172/JCI161638. PMID: 36125902Free PMC Article
Margeta M
Annu Rev Pathol 2020 Jan 24;15:261-285. Epub 2019 Oct 8 doi: 10.1146/annurev-pathmechdis-012419-032618. PMID: 31594457
Fan HC, Lee CM, Harn HJ, Cheng SN, Yuh YS
Am J Perinatol 2003 May;20(4):173-9. doi: 10.1055/s-2003-40603. PMID: 12874727

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