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).
Preview |
PDF
s00415-022-11459-3.pdf - Published Version Download (980kB) | Preview |
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 |
Archive Staff Only
 |
View Item |
