University of Pittsburgh
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.
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.
Inclusion Criteria: * Any patient with a genetic neurodegenerative disorder Exclusion Criteria: * none
Collecting information about the natural progression of these diseases
Following patients who have received HSCT as part of their clinical care.