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RecruitingNCT03333200

Longitudinal Study of Neurodegenerative Disorders

Sponsor

University of Pittsburgh

Enrollment

1,500 participants

Start Date

Jan 11, 2012

Study Type

OBSERVATIONAL

Conditions

Gaucher DiseaseBatten DiseaseLeukodystrophyALDKrabbe DiseaseMLDPelizaeus-Merzbacher DiseaseVanishing White Matter DiseaseMultiple Sulfatase Deficiency DiseaseNiemann-Pick DiseasesSandhoff DiseaseTay-Sachs DiseaseAlpha-MannosidosisMPS IMPS IIMPS IIIGM3 GangliosidosisPKANNP DeficiencyOsteopetrosisMPS IVGANGM1 GangliosidosesMorquio DiseaseS-Adenosylhomocysteine Hydrolase DeficiencyLysosomal Storage DiseasesPurine Nucleoside Phosphorylase Deficiency

Summary

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.

Eligibility

Plain Language Summary

Simplified for easier understanding

This longitudinal study follows patients who have a genetic neurodegenerative disorder over time. The goal is to understand how these diseases progress naturally, which can help inform future treatment development. You may be eligible if: - You have been diagnosed with any genetic neurodegenerative disorder You may NOT be eligible if: - There are no exclusion criteria for this study Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERPalliative Care

Collecting information about the natural progression of these diseases

BIOLOGICALHematopoetic Stem Cell Transplantation

Following patients who have received HSCT as part of their clinical care.

Locations(1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

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NCT03333200

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