NCT03333200Recruiting
Longitudinal Study of Neurodegenerative Disorders
University of Pittsburgh
Start Date
1/11/2012
Completion Date
1/1/2035
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.
Detailed 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 Criteria
Age Range: No minimum to No maximum
Inclusion Criteria:
* Any patient with a genetic neurodegenerative disorder
Exclusion Criteria:
* none
Interventions
OTHER
Palliative Care
BIOLOGICAL
Hematopoetic Stem Cell Transplantation
Conditions
MLDKrabbe DiseaseALDMPS IMPS IIMPS IIIVanishing White Matter DiseaseGM3 GangliosidosisPKANTay-Sachs DiseaseNP DeficiencyOsteopetrosisAlpha-MannosidosisSandhoff DiseaseNiemann-Pick DiseasesMPS IVGaucher DiseaseGANGM1 GangliosidosesMorquio DiseaseS-Adenosylhomocysteine Hydrolase DeficiencyBatten DiseasePelizaeus-Merzbacher DiseaseLeukodystrophyLysosomal Storage DiseasesPurine Nucleoside Phosphorylase DeficiencyMultiple Sulfatase Deficiency Disease
Locations
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania 15224
United States