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Longitudinal Study of Neurodegenerative Disorders

Recruiting
years of age
Both
Phase N/A

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Overview

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Description

Patients would be evaluated by a multidisciplinary team at specific time points every 3 months the first year, every 6 months the second year and once a year thereafter.

Eligibility

Inclusion Criteria:

  • Any patient with a genetic neurodegenerative disorder

Exclusion Criteria:

  • none

Study details

MLD, Krabbe Disease, ALD, MPS I, MPS II

NCT03333200

University of Pittsburgh

16 April 2024

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