Abstract

The purpose of this proposal is to develop new small molecule drugs for the treatment of Duchenne, and possibly other muscular dystrophies. We have chosen four targets that can alter the disease process if the protein levels are either increased or decreased. Our four targets are: utrophin (UTRN), myostatin (GDF8), the class la splice-form of insulin-like growth factor (IGF-la), and

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
5U54NS058572-05
Application #
8301741
Study Section
National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
Project Start
Project End
Budget Start
2011-07-01
Budget End
2012-06-30
Support Year
5
Fiscal Year
2011
Total Cost
$519,110
Indirect Cost

  Institution

Name
University of Pennsylvania
Department
Type
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104

  Publications

Nelson, Michael D; Rader, Florian; Tang, Xiu et al. (2014) PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy. Neurology 82:2085-91