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(A) Dose escalation regimen for adult patients administered every 2 weeks. Dose-escalation strategy for olipudase alfa after the Phase 1B study [31]. The aim of the dose-escalation process of olipudase alfa is gradual sphingomyelin debulking to prevent high ceramide release. BMI, body mass index. (B) Dose escalation regimen for pediatric patients administered every 2 weeks. Dose escalation regimen of olipudase alfa phase I/II ASCEND PEDS [27].
