Journal article icon

Journal article

Second-generation compound for the modulation of utrophin in the therapy of DMD

Abstract:

Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of the cytoskeletal protein dystrophin. There is currently no cure for DMD although various promising approaches are progressing through human clinical trials. By pharmacologically modulating the expression of the dystrophin-related protein utrophin, we have previously demonstrated in dystrophin-deficient mdx studies, daily SMT C1100 treatment significantly reduced muscle degeneration leading to impr...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


Files:
Publisher copy:
10.1093/hmg/ddv154

Authors


Expand authors...
Medical Research Council More from this funder
Publisher:
Oxford University Press Publisher's website
Journal:
Human molecular genetics Journal website
Volume:
24
Issue:
15
Pages:
4212-4224
Publication date:
2015-05-01
Acceptance date:
2015-04-27
DOI:
EISSN:
1460-2083
ISSN:
0964-6906
Pmid:
25935002
Source identifiers:
521783

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP