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It means we are relying heavily on animal data and anecdotal human reports

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One patient was reported to have learning disability with dyslexia and was also thought to be mentally retarded [5], and another had delayed psychomotor development and progressive sensory neuropathy of lower extremities, ataxia, hyperreflexia, dysarthria, and a peculiar gait suggestive of spinocerebellar degeneration [2]