Back to Trials
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

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

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