Longitudinal Study of Neurodegenerative Disorders

ConditionsMLD 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

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeNot listed

SponsorUniversity of Pittsburgh

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.

Eligibility criteria

Qualifiers

Any patient with a genetic neurodegenerative disorder

Disqualifiers

none

Trial design

Treatments tested in this trial

Palliative Care
Hematopoetic Stem Cell Transplantation

Treatment groups

No treatment groups listed

Locations

UPMC Children's Hospital of Pittsburgh15224, PittsburghPennsylvania, United States

Sponsors and collaborators

University of Pittsburgh

Lead sponsor

Source ClinicalTrials.gov