Longitudinal Studies of Brain Structure and Function in MPS Disorders

Study ID Alternative Stable ID Type
phs001328 Longitudinal

Study Description

Mucopolysaccharidosis (MPS) types I, II, IV, VI and VII, are inherited diseases that result from the bodies inability to break down large sugar molecules that are by-products of metabolism. MPS affects most organs of the body and causes abnormalities in the liver, spleen, bones, and brain. We are studying the central nervous system (primarily the brain) so we can better understand the nature of the problems individuals with MPS I, II, IV, VI and VII have so we can find ways of better treating these problems. We would like to find out about the changes in the brain of individuals with MPS I, II, IV, VI and VII using data from MRI and neuropsychological tests. This is a longitudinal study so patients who enroll will need to be seen 3 times over 5 years. The longitudinal nature of the study allows us to make conclusions about how changes in some structures of the brain and changes in cognitive ability are related.

The research objectives are:

  1. To identify abnormalities of brain (and central nervous system) structure and function in patients with MPS I, II, IV, VI and ... (Show More)

Archive Link Archive Accession
dbGaP phs001328

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