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Thesis

Mechanism of action studies of the utrophin modulator ezutromid for the treatment of Duchenne muscular dystrophy

Abstract:

Duchenne muscular dystrophy (DMD) is a muscle wasting disease arising from mutations in the dystrophin gene, affecting about 1 in 5000 boys. Whilst there is currently no available cure for DMD, a number of therapeutic strategies are under development. One such therapy aims to promote expression of utrophin, an autosomal paralogue of dystrophin. Utrophin upregulation rescues the dystrophic phenotype in disease model mice. A novel small molecule utrophin modulator, ezutromid (Summit Therapeu...

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Division:
MPLS
Department:
Chemistry
Sub department:
Organic Chemistry
Department:
University of Oxford, Department of Chemistry
Role:
Author

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Role:
Supervisor
Engineering & Physical Sciences Research Council More from this funder
Medical Research Council More from this funder
Summit Therapeutics plc More from this funder
Type of award:
DPhil
Level of award:
Doctoral
Awarding institution:
University of Oxford
Language:
English
Keywords:
Subjects:
UUID:
uuid:47554767-9235-4319-8911-5eb827081044
Deposit date:
2019-12-10

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