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Chemical proteomics and phenotypic profiling identifies the aryl hydrocarbon receptor as a molecular target of the utrophin modulator ezutromid

Abstract:

Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease arising from mutations in the dystrophin gene. Upregulation of utrophin to compensate for the missing dystrophin offers a potential therapy independent of patient genotype. The first-in-class utrophin modulator ezutromid/SMT C1100 was developed from a phenotypic screen through to a Phase 2 clinical trial. Promising efficacy and evidence of target engagement was observed in DMD patients after 24 weeks of treatment, however tri...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1002/anie.201912392

Authors


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Role:
Author
ORCID:
0000-0002-6987-3235
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Name:
Muscular Dystrophy UK
Grant:
RA4/3013/4
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Name:
Medical Research Council
Grant:
1501AV003/CA2
Publisher:
Wiley
Journal:
Angewandte Chemie More from this journal
Volume:
59
Issue:
6
Pages:
2420-2428
Publication date:
2019-11-22
Acceptance date:
2019-11-13
DOI:
EISSN:
1521-3773
ISSN:
1433-7851
Pmid:
31755636
Language:
English
Keywords:
Pubs id:
pubs:1073943
UUID:
uuid:f9724ed1-3dd0-45be-9f7e-1b9ed2e54c85
Local pid:
pubs:1073943
Source identifiers:
1073943
Deposit date:
2020-01-18

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