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.
| Condition(s) | MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niema |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| 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. |
| Who can participate | Inclusion Criteria: * Any patient with a genetic neurodegenerative disorder Exclusion Criteria: * none |
| Sex | All |
| Lead sponsor | University of Pittsburgh |
| Locations | Pittsburgh, Pennsylvania, United States |
| Start date | 2012-01-11 |
| NCT ID | NCT03333200 |
| Official listing | https://clinicaltrials.gov/study/NCT03333200 |