University of Pittsburgh
Deepa beauty Rajan
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.
MLD
Crab Disease
ALD
MPS I
MPS II
MPS III
Vanishing White Matter Disease
GM3 Gangliosidosis
Pk
Tay-Sachs Disease
NP Deficiency
Osteopetrosis
Alpha-Mannosidosis
Sandhoff Disease
Niemann-Pick Diseases
MPS IV
Gaucher Disease
Both
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
Palliative Care
Hematopoetic Stem Cell Transplantation
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.
| Study Type : | OBSERVATIONAL |
| Estimated Enrollment : | 1500 participants |
| Official Title : | Longitudinal Study of Neurodegenerative Disorders |
| Actual Study Start Date : | 2012-01-11 |
| Estimated Primary Completion Date : | 2030-01 |
| Estimated Study Completion Date : | 2035-01 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
RECRUITING
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224