Inclusion body myositis: from genetics to clinical trials.
Clinical trials
Gene therapy
Genetic susceptibility
Hereditary inclusion body myositis
Inflammation
Neurodegeneration
Sporadic inclusion body myositis
Journal
Journal of neurology
ISSN: 1432-1459
Titre abrégé: J Neurol
Pays: Germany
ID NLM: 0423161
Informations de publication
Date de publication:
Mar 2023
Mar 2023
Historique:
received:
17
10
2022
accepted:
25
10
2022
pubmed:
19
11
2022
medline:
3
3
2023
entrez:
18
11
2022
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 36399165
doi: 10.1007/s00415-022-11459-3
pii: 10.1007/s00415-022-11459-3
pmc: PMC9971047
doi:
Types de publication
Review
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
1787-1797Subventions
Organisme : Medical Research Council
ID : MR/S01165X/1
Pays : United Kingdom
Organisme : Medical Research Council Centre for Medical Mycology
ID : MR/S01165X/1
Organisme : Medical Research Council Centre for Medical Mycology
ID : MR/S005021/1
Organisme : Medical Research Council Centre for Medical Mycology
ID : G0601943
Informations de copyright
© 2022. The Author(s).
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