Taking people in United StatesNo phase
Following rare genetic brain disorders over time in patients
Observes how rare inherited brain disorders progress, including effects of treatments.
Plain-language title generated from the registry record
Registry title: Longitudinal Study of Neurodegenerative Disorders
Key facts
- Where
- Pittsburgh, United StatesSee the site
Where in United States
- UPMC Children's Hospital of PittsburghPittsburgh, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- University of Pittsburgh
- Trial code
- NCT03333200
- Record updated
- 9 February 2026
- Original description
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.Read full original descriptionShow less
Longitudinal Study of Neurodegenerative Disorders
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.
Inclusion: Any patient with a genetic neurodegenerative disorder.
Exclusion: none.