The overall aim of this project is to expand our knowledge of the clinical features of MPS NIC and MPS HID disease by carrying out a prospective natural history study. We have identified 15 patients with MPS INC and 2 patients with MPSIIID. During the course of the study, we will determine the genotype and the residual enzyme activity, as well as do periodic measurements by neurocognitive testing, measure glycosaminoglycans in urine, observations of clinical status, and use brain imaging to document the course of the disease. Our hypothesis is that disease progression will be manifested by neurocognitive decline, and will correlate with genotype and/or residual enzyme activity, as well as with an increase in glycosaminoglycan levels in urine.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
2U54NS065768-06
Application #
8907075
Study Section
Special Emphasis Panel (ZTR1-CI-8 (01))
Program Officer
Morris, Jill A
Project Start
Project End
Budget Start
2014-09-30
Budget End
2015-07-31
Support Year
6
Fiscal Year
2014
Total Cost
$7,615
Indirect Cost
$2,605
Name
University of Minnesota Twin Cities
Department
Type
DUNS #
555917996
City
Minneapolis
State
MN
Country
United States
Zip Code
55455
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