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Inclusion body myositis: from genetics to clinical trials

Nagy, Sara; Khan, Alaa; Machado, Pedro M; Houlden, Henry; (2022) Inclusion body myositis: from genetics to clinical trials. Journal of Neurology 10.1007/s00415-022-11459-3. (In press). Green open access

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Abstract

Inclusion body myositis (IBM) belongs to the group of idiopathic inflammatory myopathies and is characterized by a slowly progressive disease course with asymmetric muscle weakness of predominantly the finger flexors and knee extensors. The disease leads to severe disability and most patients lose ambulation due to lack of curative or disease-modifying treatment options. Despite some genes reported to be associated with hereditary IBM (a distinct group of conditions), data on the genetic susceptibility of sporadic IBM are very limited. This review gives an overview of the disease and focuses on the current genetic knowledge and potential therapeutic implications.

Type: Article
Title: Inclusion body myositis: from genetics to clinical trials
Location: Germany
Open access status: An open access version is available from UCL Discovery
DOI: 10.1007/s00415-022-11459-3
Publisher version: https://doi.org/10.1007/s00415-022-11459-3
Language: English
Additional information: © 2022 Springer Nature Switzerland AG. This article is licensed under a Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/).
Keywords: Clinical trials, Gene therapy, Genetic susceptibility, Hereditary inclusion body myositis, Inflammation, Neurodegeneration, Sporadic inclusion body myositis
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases
URI: https://discovery.ucl.ac.uk/id/eprint/10160188
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