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In trials, patients taking this drug shed an average of 24% of their body weight

Cell-free supernatants were collected and stored at -20C for the future assays and granulomas were harvested by adding ice-cold sterile 1x PBS to wells, followed by gentle scraping to achieve maximum recovery of granuloma lysates and stored at -20C

R., De Jonghe, B

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)