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The HOPE study showed an increase in hemoglobin levels and reduced markers of hemolysis in 274 patients with HbS that were randomly assigned to receive the study drug versus placebo

Zhang C, Liu Z, Wang F, Zhang B, Zhang X, Guo P, et al

Long-term safety endpoints had the primarily purpose of excluding the presence of infections due the possible non-sterility of the cell product (a risk minimized by the extensive in vitro examinations), tumor formation (risk minimized by the in vivo evidence obtained by infusion of supra maximal doses of cells in immunosuppressed mice) as well as changes in neurological status that would not be expected based on the disease course

Gently swirl (do not shake vigorously) until the powder is fully dissolved