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The smaller rare disease patient population, and very often the heterogeneity of disease progression seen with many rare diseases, can seriously impact the ability to establish evidence of effectiveness using conventional trial designs

The formation of GSH-conjugates is involved in electrophile detoxification, and thus higher levels of glutathione might prevent these reactive metabolites from reacting with and damaging cellular macromolecules 21

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However, preclinical studies indicate that while DFP improves motor function in animal models, it does not significantly reduce -syn aggregation [80]