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Myopathy with tubular aggregates(TAM)

MedGen UID:
98050
Concept ID:
C0410207
Disease or Syndrome
Synonyms: TAM; Tubular Aggregate Myopathy
SNOMED CT: Myopathy with tubular aggregates (240087000)
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.
 
Monarch Initiative: MONDO:0008051
OMIM®: 160565
OMIM® Phenotypic series: PS160565
Orphanet: ORPHA2593

Definition

A rare congenital myopathy characterized ultrastructurally by the presence of tubular aggregates in the subsarcolemmal region of the muscle fiber. It most commonly presents with slowly progressive proximal muscle weakness predominantly of the lower limbs, periodic paralysis, post-exertion muscle cramps, and muscular pain. Ocular anomalies like ophthalmoplegia or pupillary abnormalities may be associated. The intensity of the symptoms is variable, cases with normal muscle strength but myalgia or fatigue, as well as clinically asymptomatic cases have been described. [from ORDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVMyopathy with tubular aggregates
Follow this link to review classifications for Myopathy with tubular aggregates in Orphanet.

Professional guidelines

PubMed

Ramos-Fransi A, Martínez-Piñeiro A, Almendrote M, Lucente G, Carrato C, Ballester-Lopez A, Lucia A, Pintos-Morell G, Nogales-Gadea G, Coll-Cantí J
Muscle Nerve 2018 Jun;57(6):E138-E140. Epub 2018 Feb 2 doi: 10.1002/mus.26078. PMID: 29350769
Lehmann-Horn F, Jurkat-Rott K, Rüdel R; Ulm Muscle Centre
Acta Myol 2008 Dec;27(3):98-113. PMID: 19472919Free PMC Article
Marston SB, Hodgkinson JL
J Muscle Res Cell Motil 2001;22(1):1-4. doi: 10.1023/a:1010355716511. PMID: 11563546

Recent clinical studies

Etiology

Vattemi GNA, Rossi D, Galli L, Catallo MR, Pancheri E, Marchetto G, Cisterna B, Malatesta M, Pierantozzi E, Tonin P, Sorrentino V
Eur J Neurosci 2022 Aug;56(3):4214-4223. Epub 2022 Jun 13 doi: 10.1111/ejn.15728. PMID: 35666680Free PMC Article
Finsterer J, Scorza FA, Scorza CA
J Clin Neuromuscul Dis 2019 Dec;21(2):90-102. doi: 10.1097/CND.0000000000000269. PMID: 31743252
Filosto M, Tonin P, Vattemi G, Bertolasi L, Simonati A, Rizzuto N, Tomelleri G
Neurology 2007 Jan 16;68(3):181-6. doi: 10.1212/01.wnl.0000252252.29532.cc. PMID: 17224570
Alonso-Losada G, Cimas I, Pego R, La Torre P, Teijeira S, Navarro C
Clin Neuropathol 1998 Jan-Feb;17(1):50-4. PMID: 9496541
Tulinius MH, Lundberg A, Oldfors A
Pediatr Neurol 1996 Jul;15(1):68-71. doi: 10.1016/0887-8994(96)00094-x. PMID: 8858706

Diagnosis

Jiang K, Zheng Y, Lin J, Wu X, Yu Y, Zhu M, Fang X, Zhou M, Li X, Hong D
Brain Behav 2022 Feb;12(2):e2469. Epub 2022 Jan 3 doi: 10.1002/brb3.2469. PMID: 34978387Free PMC Article
Madigan NN, Liewluck T, Milone M, Naddaf E
Neurology 2019 Aug 13;93(7):313-314. doi: 10.1212/WNL.0000000000007952. PMID: 31405936
Li A, Kang X, Edelman F, Waclawik AJ
J Child Neurol 2019 May;34(6):321-324. Epub 2019 Feb 14 doi: 10.1177/0883073819829389. PMID: 30761937
Onda A, Miyagawa S, Takahashi N, Gochi M, Takagi M, Nishino I, Suzuki S, Oishi C, Yaguchi H
Intern Med 2019 Jun 1;58(11):1635-1638. Epub 2019 Feb 1 doi: 10.2169/internalmedicine.1956-18. PMID: 30713313Free PMC Article
Korényi-Both A, Korényi-Both I
J Med 1987;18(2):93-107. PMID: 3323392

Therapy

Onda A, Miyagawa S, Takahashi N, Gochi M, Takagi M, Nishino I, Suzuki S, Oishi C, Yaguchi H
Intern Med 2019 Jun 1;58(11):1635-1638. Epub 2019 Feb 1 doi: 10.2169/internalmedicine.1956-18. PMID: 30713313Free PMC Article
Lanzi R, Previtali SC, Sansone V, Scavini M, Fortunato M, Gatti E, Meola G, Bosi E, Losa M
J Endocrinol Invest 2007 Apr;30(4):341-5. doi: 10.1007/BF03346302. PMID: 17556873
Palmucci L, Doriguzzi C, Anzil AP
J Neurol 1985;232(6):374-7. doi: 10.1007/BF00313840. PMID: 4078604

Prognosis

Jiang K, Zheng Y, Lin J, Wu X, Yu Y, Zhu M, Fang X, Zhou M, Li X, Hong D
Brain Behav 2022 Feb;12(2):e2469. Epub 2022 Jan 3 doi: 10.1002/brb3.2469. PMID: 34978387Free PMC Article

Clinical prediction guides

Jiang K, Zheng Y, Lin J, Wu X, Yu Y, Zhu M, Fang X, Zhou M, Li X, Hong D
Brain Behav 2022 Feb;12(2):e2469. Epub 2022 Jan 3 doi: 10.1002/brb3.2469. PMID: 34978387Free PMC Article
Lanzi R, Previtali SC, Sansone V, Scavini M, Fortunato M, Gatti E, Meola G, Bosi E, Losa M
J Endocrinol Invest 2007 Apr;30(4):341-5. doi: 10.1007/BF03346302. PMID: 17556873
Filosto M, Tonin P, Vattemi G, Bertolasi L, Simonati A, Rizzuto N, Tomelleri G
Neurology 2007 Jan 16;68(3):181-6. doi: 10.1212/01.wnl.0000252252.29532.cc. PMID: 17224570

Recent systematic reviews

Finsterer J, Scorza FA, Scorza CA
J Clin Neuromuscul Dis 2019 Dec;21(2):90-102. doi: 10.1097/CND.0000000000000269. PMID: 31743252

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