Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Inherited Disease 2-Hydroxyglutaricaciduria Adrenal Gland Diseases Adrenal Insufficiency Adrenoleukodystrophy Alexander Disease Basal Ganglia Diseases Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cysts Demyelinating Diseases Digestive System Diseases Disease Attributes Endocrine System Diseases Fahr's disease Genetic Diseases, Inborn Genetic Diseases, X-Linked Hepatolenticular Degeneration Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Intellectual Disability Leukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Leukoencephalopathies Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Liver Diseases Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Disease Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Methylmalonic acidemia with homocystinuria Movement Disorders Neoplasms Nervous System Diseases Neuro-Degenerative Disease Neurobehavioral Manifestations Neurodegenerative Diseases Neurologic Disorder Neurologic Manifestations Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Pelizaeus-Merzbacher Disease Peroxisomal Disorders Polycystic lipomembranous osteodysplasia with sclerosing leukoencephalopathy Rare Diseases Sphingolipidoses Sulfatidosis Undiagnosed Disease Undiagnosed Diseases X-Linked Intellectual Disability Xanthomatosis Xanthomatosis, Cerebrotendinous