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That rationale must be backed by non-clinical data confirming the compound behaves as expected and by a thorough safety and toxicology program that identifies potential risks and establishes a safe starting dose for the first human administration and the maximum human exposure that should not be surpassed

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The most prevalent gene-associated PD risk factor is the catabolism of the glycolipid glucocerebroside into glucose and ceramide in the lysosome [227,228], which causes Gaucher disease

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