Longitudinal Study of Neurodegenerative Disorders
Recruiting
Observational Study
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
Niemann-Pick Diseases
MPS IV
Gaucher Disease
GAN
GM1 Gangliosidoses
Morquio Disease
S-Adenosylhomocysteine Hydrolase Deficiency
Batten Disease
Pelizaeus-Merzbacher Disease
Leukodystrophy
Lysosomal Storage Diseases
Purine Nucleoside Phosphorylase Deficiency
Multiple Sulfatase Deficiency Disease
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 1,500 (estimated)
- Sponsor
- University of Pittsburgh · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Any patient with a genetic neurodegenerative disorder
Exclusion Criteria:
* none
Contacts