This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The purpose of this study is to learn about the safety and effectiveness of a new drug, called Myozyme, used to treat infantile-onset Pompe disease. In infantile Pompe Disease, too much glycogen builds up and is stored in certain body tissues, especially heart, skeletal muscle, and liver tissues. This prevents the tissues from performing their normal functions. This causes the heart to enlarge (cardiomegaly) and its walls to thicken (hypertrophic cardiomyopathy), which causes less pumping of blood (heart failure). Every 2 weeks, 16 patients worldwide will be given an intravenous infusion (IV) of Myozyme, the enzyme that is missing in the bodies of persons with Pompe disease. The children will be followed closely to see how the disease and the study medicine affects the body, health, and the child's ability to grow and develop motor skills.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR008084-13
Application #
7374543
Study Section
Special Emphasis Panel (ZRR1-CR-9 (01))
Project Start
2005-12-01
Project End
2006-11-30
Budget Start
2005-12-01
Budget End
2006-11-30
Support Year
13
Fiscal Year
2006
Total Cost
$5,316
Indirect Cost
Name
Children's Hospital Med Ctr (Cincinnati)
Department
Type
DUNS #
071284913
City
Cincinnati
State
OH
Country
United States
Zip Code
45229
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