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 ILysosomal Storage DiseasesMPS IIMPS IIIGM3 GangliosidosisPKANNP DeficiencyOsteopetrosisMPS IVGANGM1 GangliosidosesMorquio DiseaseS-Adenosylhomocysteine Hydrolase DeficiencyPurine 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
Inclusion Criteria1
- Any patient with a genetic neurodegenerative disorder
Exclusion Criteria1
- none
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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)
View Full Details on ClinicalTrials.gov
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NCT03333200
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