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Results of the study showed an 84% event-free survival rate after four years compared with 73% in an external control arm
Study design We employed a target trial design to emulate a RCT using observational data (Supplementary Table 1)
Notably, they have announced a self-pay price point of roughly $149 per month with savings offers for the starting dose
On day 19 of admission, the infant, now generally in good condition with the metabolic acidosis corrected, was discharged
Independent replication by unaffiliated laboratories is limited
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