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TPM3-related myopathy

MedGen UID:
1001695
Concept ID:
CN323193
Disease or Syndrome
Synonyms: congenital myopathy related to TPM3; TPM3 myopathy
 
Monarch Initiative: MONDO:0100108

Definition

TPM3-related myopathy is a disorder of the musculoskeletal system that covers a wide spectrum of phenotypes and is caused by pathogenic variants in the skeletal muscle ?-Tropomyosin gene. These variants lead to a variety of overlapping adult onset and congenital myopathies characterized by muscle weakness, hypotonia, motor delay, myopathic facies, scoliosis, and sometimes respiratory involvement. Histologic findings on skeletal muscle biopsy are variable with nemaline and intranuclear bodies, cap-like lesions, fiber-type disproportion, and dystrophic features even in patients with the same mutation. [from MONDO]

Recent clinical studies

Etiology

Donkervoort S, Papadaki M, de Winter JM, Neu MB, Kirschner J, Bolduc V, Yang ML, Gibbons MA, Hu Y, Dastgir J, Leach ME, Rutkowski A, Foley AR, Krüger M, Wartchow EP, McNamara E, Ong R, Nowak KJ, Laing NG, Clarke NF, Ottenheijm C, Marston SB, Bönnemann CG
Ann Neurol 2015 Dec;78(6):982-994. Epub 2015 Nov 13 doi: 10.1002/ana.24535. PMID: 26418456Free PMC Article

Diagnosis

Donkervoort S, Papadaki M, de Winter JM, Neu MB, Kirschner J, Bolduc V, Yang ML, Gibbons MA, Hu Y, Dastgir J, Leach ME, Rutkowski A, Foley AR, Krüger M, Wartchow EP, McNamara E, Ong R, Nowak KJ, Laing NG, Clarke NF, Ottenheijm C, Marston SB, Bönnemann CG
Ann Neurol 2015 Dec;78(6):982-994. Epub 2015 Nov 13 doi: 10.1002/ana.24535. PMID: 26418456Free PMC Article
Schreckenbach T, Schröder JM, Voit T, Abicht A, Neuen-Jacob E, Roos A, Bulst S, Kuhl C, Schulz JB, Weis J, Claeys KG
Neuromuscul Disord 2014 Feb;24(2):117-24. Epub 2013 Oct 23 doi: 10.1016/j.nmd.2013.10.002. PMID: 24239060

Prognosis

Schreckenbach T, Schröder JM, Voit T, Abicht A, Neuen-Jacob E, Roos A, Bulst S, Kuhl C, Schulz JB, Weis J, Claeys KG
Neuromuscul Disord 2014 Feb;24(2):117-24. Epub 2013 Oct 23 doi: 10.1016/j.nmd.2013.10.002. PMID: 24239060

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