Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies

P Soblechero‐Martín, A López‐Martínez… - Neuropathology and …, 2021 - Wiley Online Library
… SMT022357, is a second-generation compound structurally related to ezutromid, sharing
the same mechanism of action but with improved physicochemical properties and a more …

2-Arylbenzo[d]oxazole Phosphinate Esters as Second-Generation Modulators of Utrophin for the Treatment of Duchenne Muscular Dystrophy

A Babbs, A Berg, M Chatzopoulou… - Journal of Medicinal …, 2020 - ACS Publications
Utrophin modulation is a promising therapeutic strategy for Duchenne muscular dystrophy
(DMD), … The ability of the compounds to increase utrophin levels was investigated using the …

[HTML][HTML] Structure-activity relationships of 2-pyrimidinecarbohydrazides as utrophin modulators for the potential treatment of Duchenne muscular dystrophy

M Chatzopoulou, D Conole, E Emer, JA Rowley… - Bioorganic & Medicinal …, 2022 - Elsevier
… insights regarding utrophin upregulation as a strategy for DMD therapeutic intervention. …
the second generation phosphinates. Compound 1 was also found to increase utrophin mRNA …

Discovery of Quinazoline and Quinoline-Based Small Molecules as Utrophin Upregulators via AhR Antagonism for the Treatment of Duchenne Muscular Dystrophy

S Ghosh, MU Arshi, S Ghosh, M Jash… - Journal of Medicinal …, 2024 - ACS Publications
… of another second-generation utrophin modulator SMT022357 … candidacy of SG-02 for DMD
therapy. Our data suggest that … lead compounds exhibited a significant elevation in utrophin

Duchenne muscular dystrophy: pathogenesis and promising therapies

M Chang, Y Cai, Z Gao, X Chen, B Liu, C Zhang… - Journal of …, 2023 - Springer
… Furthermore, GA-binding protein can enhance utrophin promoter activity … Therefore,
therapeutic strategies that modulate the … ) are a class of compounds that function by inhibiting the …

Drug development progress in duchenne muscular dystrophy

J Deng, J Zhang, K Shi, Z Liu - Frontiers in Pharmacology, 2022 - frontiersin.org
therapies, exon skipping, and readthrough therapy, as well as strategies that target secondary
pathology of DMD, such as novel anti-inflammatory compounds, … of utrophin appeared to …

Innovative therapeutic approaches for Duchenne muscular dystrophy

F Fortunato, R Rossi, MS Falzarano… - Journal of Clinical …, 2021 - mdpi.com
second-generation drugs now entering in clinical trials as gene … , utrophin modulation
remains a promising therapeutic … Anti-inflammatory factors, utrophin upregulators, compounds/…

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
… genes such as utrophin and combination therapies, are also … currently exon skipping, whereby
modulation of splicing is used … 3d), with the leading compound AOC 1044, targeting DMD

Dystrophin-and utrophin-based therapeutic approaches for treatment of duchenne muscular dystrophy: a comparative review

S Szwec, Z Kapłucha, JS Chamberlain, P Konieczny - BioDrugs, 2024 - Springer
utrophin domains in Duchenne muscular dystrophy treatment, providing insights into designing
novel therapeutic compounds … with multiple roles in modulating various cell functions, …

The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy

S Guiraud, B Edwards, A Babbs… - Human Molecular …, 2019 - academic.oup.com
… Transgenic mice overexpressing utrophin showed its localization … of utrophin-based therapies
applicable to all DMD patients … that utrophin-based strategies such as utrophin modulation