Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
NCT02292654
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clinicaltrials.gov ↗
PHASE1,PHASE2
Phase
COMPLETED
Status
20
Enrollment
INDUSTRY
Sponsor class
Conditions
Sphingomyelin Lipidosis
Interventions
DRUG:
Olipudase alfa
Sponsor
Genzyme, a Sanofi Company