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Supplementation with SA delivered by aceneuramic acid extended release stabilized muscle strength in GNE myopathy patients 7 , however, this study did not demonstrate a statistically significant difference in the muscle strength of patients compared to placebo and hence, discontinued from clinical trials (Ultragenyx, 2017)

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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