Longitudinal Study of Neurodegenerative Disorders
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- STATUS
- Recruiting
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- End date
- Jan 13, 2035
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- participants needed
- 1500
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- sponsor
- University of Pittsburgh
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.
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.
Details
| Condition | S-Adenosylhomocysteine Hydrolase Deficiency, Adrenoleukodystrophy, mucopolysaccharidosis type i, Metachromatic leukodystrophy, Batten Disease, Hunter's Syndrome, hurler syndrome, GM1 Gangliosidoses, Hallervorden-Spatz Disease, niemann pick disease, Pelizaeus-Merzbacher Disease, Metachromatic Leukodystrophy - MLD, Tay-Sachs Disease, Giant Axonal Neuropathy, gaucher's disease, dysostosis multiplex, Hunter Syndrome (MPS II), tay sachs disease, Neuronal Ceroid Lipofuscinoses (NCL), GM3 Gangliosidosis, Multiple Sulfatase Deficiency, krabbe disease, Osteopetrosis, Infantile Neuronal Ceroid Lipofuscinosis, Gaucher Disease, Sandhoff Disease, Hurler's Syndrome, Neuronal ceroid lipofuscinosis, Krabbe's Disease, Sanfilippo's Syndrome, mps iii, Lysosomal Storage Disease, NP Deficiency, Purine Nucleoside Phosphorylase Deficiency, Niemann-Pick Disease, alpha-Mannosidosis, Leukodystrophy, Osteochondrodystrophy, Vanishing White Matter Disease |
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| Treatment | Palliative Care, Hematopoetic Stem Cell Transplantation |
| Clinical Study Identifier | NCT03333200 |
| Sponsor | University of Pittsburgh |
| Last Modified on | 13 November 2021 |
How to participate?
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
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