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A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

2010-07-15 17:00:00 | BioPortfolio

Summary

The purpose is to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, development, and imaging measures.

Description

The purpose of this multicenter longitudinal, prospective, observational, natural history study of patients with MPS IIIA is to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, development, and imaging measures.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Conditions

Sanfilippo Syndrome Type A

Intervention

assessment

Location

University of Minnesota
Minneapolis
Minnesota
United States
55455

Status

Recruiting

Source

Shire Human Genetic Therapies, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2010-07-15T17:00:00-0400

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Medical and Biotech [MESH] Definitions

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