Longitudinal Study of Neurodegenerative Disorders

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
• BIOLOGICAL: Hematopoetic Stem Cell Transplantation

Sponsor

University of Pittsburgh