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Upon internalization, pyrophosphodiester passes through the endosome-lysosome pathway, which undergoes a two-step enzymatic linker cleavage, releasing first the payload-monophosphate molecule, followed by the free payload

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Exercise capacity, measured by the 6-minute walk test, showed that 50% of ARA-290 patients could walk at least 25 meters more compared with only 12% of placebo patients