Longitudinal Study of Neurodegenerative Disorders

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.

Conditions

• 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

Interventions

OTHER

Palliative Care

Collecting information about the natural progression of these diseases

BIOLOGICAL

Hematopoetic Stem Cell Transplantation

Following patients who have received HSCT as part of their clinical care.

Sponsors & Collaborators

• University of Pittsburgh

  lead OTHER

Eligibility

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-01-11

Primary Completion

2030-01-31

Completion

2035-01-31

Countries

• United States

Study Locations