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Longitudinal Study of Neurodegenerative Disorders

Longitudinal Study of Neurodegenerative Disorders

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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
    MPS III
    Vanishing White Matter Disease
    GM3 Gangliosidosis
    PKAN
    Tay-Sachs Disease
    NP Deficiency
    Osteopetrosis
    Alpha-Mannosidosis
    Sandhoff Disease
    Niemann-Pick Diseases
    MPS IV
    Gaucher Disease
    GAN
    GM1 Gangliosidoses
    Morquio Disease
    S-Adenosylhomocysteine Hydrolase Deficiency
    Batten Disease
    Pelizaeus-Merzbacher Disease
    Leukodystrophy
    Lysosomal Storage Diseases
    Purine Nucleoside Phosphorylase Deficiency
    Multiple Sulfatase Deficiency Disease

NCT03333200

University of Pittsburgh

16 April 2024

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