Sphingomyelin Lipidosis

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CLINICAL_TRIALS trial_result · 2021-02-01
Clinical Trial Update: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency [Phase 1, Phase 2]
Phase 1/2 trial assessing repeat ascending doses of olipudase alfa in pediatric patients under 18 with acid sphingomyelinase deficiency; saf…