Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy.
Animals
Biomarkers
Clinical Studies as Topic
Combined Modality Therapy
Disease Management
Disease Susceptibility
Drug Development
Drug Evaluation, Preclinical
Genetic Predisposition to Disease
Homeodomain Proteins
/ genetics
Humans
Molecular Targeted Therapy
Muscular Dystrophy, Facioscapulohumeral
/ diagnosis
Oxidation-Reduction
Treatment Outcome
DUX4
anabolic
clinical outcome measures
clinical trials
facioscapulohumeral muscular dystrophy
therapies
Journal
Trends in molecular medicine
ISSN: 1471-499X
Titre abrégé: Trends Mol Med
Pays: England
ID NLM: 100966035
Informations de publication
Date de publication:
02 2021
02 2021
Historique:
received:
14
07
2020
revised:
14
09
2020
accepted:
15
09
2020
pubmed:
24
10
2020
medline:
16
10
2021
entrez:
23
10
2020
Statut:
ppublish
Résumé
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common types of muscular dystrophy, affecting roughly one in 8000 individuals. The complex underlying genetics and poor mechanistic understanding has caused a bottleneck in therapeutic development. Until the discovery of DUX4 and its causal role in FSHD, most trials were untargeted with limited results. Emerging approaches can learn from these early trials to increase their chance of success. Here, we explore the evolution of FSHD clinical trials from nonspecific anabolic or anti-inflammatory/oxidant strategies to cutting-edge molecular therapies targeting DUX4, and we discuss the importance of clinical outcome measures. With combined advances across multiple facets of FSHD research, the field is now poised to accelerate the process of therapeutic discovery and testing.
Identifiants
pubmed: 33092966
pii: S1471-4914(20)30239-2
doi: 10.1016/j.molmed.2020.09.008
pmc: PMC8048701
mid: NIHMS1688257
pii:
doi:
Substances chimiques
Biomarkers
0
DUX4L1 protein, human
0
Homeodomain Proteins
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
123-137Subventions
Organisme : NICHD NIH HHS
ID : P50 HD060848
Pays : United States
Informations de copyright
Copyright © 2020 Elsevier Ltd. All rights reserved.
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