An update on Becker muscular dystrophy.
Journal
Current opinion in neurology
ISSN: 1473-6551
Titre abrégé: Curr Opin Neurol
Pays: England
ID NLM: 9319162
Informations de publication
Date de publication:
01 10 2023
01 10 2023
Historique:
medline:
11
9
2023
pubmed:
18
8
2023
entrez:
17
8
2023
Statut:
ppublish
Résumé
The purpose of this review is to summarise the recent developments in trial readiness, natural history studies, and interventional clinical trials for Becker muscular dystrophy (BMD). As several treatment concepts have claimed to convert patients with Duchenne muscular dystrophy (DMD) into a BMD phenotype, BMD itself has moved into the focus of clinical research. Natural history studies have helped to better characterize patients with BMD and the disease is now a target for interventional trials. In parallel, there have been advances in diagnostics and in the development of preclinical models. Despite increased collaborative efforts to improve trial readiness amongst patients with BMD, there is still a lack of long-term natural history data, and the broad spectrum of disease severity remains a challenge for well designed clinical trials.
Identifiants
pubmed: 37591308
doi: 10.1097/WCO.0000000000001191
pii: 00019052-990000000-00093
pmc: PMC10487383
doi:
Banques de données
ClinicalTrials.gov
['NCT05257473']
Types de publication
Review
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
450-454Subventions
Organisme : Department of Health
Pays : United Kingdom
Informations de copyright
Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc.
Références
BMC Med Genomics. 2023 May 30;16(1):121
pubmed: 37254189
N Engl J Med. 1988 May 26;318(21):1363-8
pubmed: 3285207
J Neuromuscul Dis. 2022;9(2):275-287
pubmed: 35124660
Front Neurol. 2023 Jan 30;14:1095121
pubmed: 36793492
Front Neurol. 2023 Feb 01;14:1107474
pubmed: 36816559
Genet Med. 2020 Sep;22(9):1478-1488
pubmed: 32528171
Mol Genet Genomic Med. 2021 Sep;9(9):e1759
pubmed: 34327855
Front Genet. 2022 Aug 25;13:979732
pubmed: 36092865
Lancet Neurol. 2018 Mar;17(3):251-267
pubmed: 29395989
Neurol Genet. 2020 Dec 24;7(1):e536
pubmed: 33376799
Ann Clin Transl Neurol. 2018 Dec 01;5(12):1574-1587
pubmed: 30564623
Front Neurol. 2021 Aug 05;12:714677
pubmed: 34421809
JAMA Neurol. 2022 Oct 1;79(10):1005-1014
pubmed: 36036925
J Neuromuscul Dis. 2021;8(4):525-535
pubmed: 33843695
Sci Transl Med. 2012 Nov 28;4(162):162ra155
pubmed: 23197572
BMC Med Genomics. 2021 Jul 8;14(1):181
pubmed: 34238289
Muscle Nerve. 2021 Feb;63(2):239-249
pubmed: 33125736
Hum Mutat. 2021 May;42(5):626-638
pubmed: 33644936
Lancet Neurol. 2018 May;17(5):445-455
pubmed: 29398641
Appl Clin Genet. 2021 Oct 01;14:399-408
pubmed: 34629887
Sci Rep. 2016 Sep 01;6:32439
pubmed: 27582364
Amino Acids. 2018 Oct;50(10):1391-1406
pubmed: 30003335
Muscle Nerve. 2020 Sep;62(3):369-376
pubmed: 32564389
Lancet Neurol. 2018 Apr;17(4):347-361
pubmed: 29395990
Ann Transl Med. 2021 May;9(9):766
pubmed: 34268379
Proc Natl Acad Sci U S A. 2008 Dec 9;105(49):19183-7
pubmed: 19047631
J Cachexia Sarcopenia Muscle. 2023 Apr;14(2):940-954
pubmed: 36628607
eNeurologicalSci. 2023 Jan 26;30:100446
pubmed: 36845278
Dis Model Mech. 2020 Sep 28;13(9):
pubmed: 32859695
Neurology. 2021 Aug 3;97(5):e513-e522
pubmed: 34162720
J Neuromuscul Dis. 2021;8(4):495-502
pubmed: 33814458
J Pers Med. 2020 Sep 03;10(3):
pubmed: 32899151
Int J Mol Sci. 2023 Feb 21;24(5):
pubmed: 36901738