Longitudinal Study of Neurodegenerative Disorders

Status Recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

ALD
Alpha-Mannosidosis
Batten Disease
Deficiency Diseases
GAN
Gangliosidoses
Gangliosidosis, GM1
Gaucher Disease
GM1 Gangliosidoses
GM3 Gangliosidosis
Krabbe Disease
Leukodystrophy
Leukodystrophy, Globoid Cell
Leukoencephalopathies
Lysosomal Storage Diseases
MLD
Morquio Disease
MPS I
MPS II
MPS III
MPS IV
Mucopolysaccharidosis IV
Multiple Sulfatase Deficiency Disease
Neurodegenerative Diseases
Neuronal Ceroid-Lipofuscinoses
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
NP Deficiency
Osteopetrosis
Pelizaeus-Merzbacher Disease
Pick Disease of the Brain
PKAN
Purine Nucleoside Phosphorylase Deficiency
S-Adenosylhomocysteine Hydrolase Deficiency
Sandhoff Disease
Tay-Sachs Disease
Vanishing White Matter Disease

Clinical Trial Details

NCT number	NCT03333200
Study type	Observational
Source	University of Pittsburgh
Contact	Deepa Rajan, MD
Phone	412-692-8388
Email	rajands@upmc.edu
Status	Recruiting
Phase
Start date	January 11, 2012
Completion date	January 2035

View Details

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