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Canavan disease Synucleinopathy Friedreich's ataxia Sanfilippo syndrome Leigh syndrome Amyotrophic lateral sclerosis Alexander disease Familial dysautonomia Huntington's disease Progressive muscular atrophy Metachromatic leukodystrophy Aa amyloidosis Glycogen storage disease type ii Melas syndrome Spinal and bulbar muscular atrophy Lafora disease Refsum disease Multiple system atrophy Pyruvate dehydrogenase deficiency Glycogen storage disease type iii Neurodegenerative disease Alkaptonuria Neuronal ceroid lipofuscinosis I-cell disease Granulomatosis with polyangiitis Patisiran Familial hypercholesterolemia Myotonic dystrophy Menkes disease Spinocerebellar ataxia Opsoclonus myoclonus syndrome Binswanger's disease Tauopathy Stickler syndrome Cancer syndrome Tuberous sclerosis Gaucher's disease Hyperkalemic periodic paralysis Congenital myasthenic syndrome Krabbe disease Spinal muscular atrophy Methylmalonic acidemia Familial adenomatous polyposis Al amyloidosis Neurofibromatosis type ii Severe combined immunodeficiency Cystinuria Mixed connective tissue disease Mcleod syndrome Cerebral amyloid angiopathy Hypokalemic periodic paralysis Motor neuron disease Canine degenerative myelopathy Fabry disease Hashimoto's encephalopathy Muscular dystrophy Hartnup disease Axial spondyloarthritis Antisynthetase syndrome Glycogen storage disease type v Cockayne syndrome Neurosarcoidosis Homocystinuria Parkinson's disease Arrhythmogenic cardiomyopathy Cadasil Degenerative disc disease Idiopathic pulmonary fibrosis Becker muscular dystrophy Corticobasal degeneration Notalgia paresthetica Corneal dystrophy Tyrosinemia type i Gardner's syndrome Hypophosphatasia Cardiac amyloidosis Dysplastic nevus syndrome Posterior cortical atrophy Primary ciliary dyskinesia Acdc Ipex syndrome Rasmussen's encephalitis Pseudocholinesterase deficiency Amyloidosis Lysosomal acid lipase deficiency Susac's syndrome Dyskeratosis congenita Alpha-1 antitrypsin deficiency Hyperimmunoglobulin e syndrome Parkinson-plus syndrome Abetalipoproteinemia Diastrophic dysplasia Systemic scleroderma Glycogen storage disease type i Arteriosclerosis obliterans Addison's disease Lateral medullary syndrome Familial dysbetalipoproteinemia Mucolipidosis Athetoid cerebral palsy

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