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]

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Summary

Phase 1/2 trial assessing repeat ascending doses of olipudase alfa in pediatric patients under 18 with acid sphingomyelinase deficiency; safety, tolerability, p…

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Diseases / indications

• Acid Sphingomyelinase Deficiency
• Sphingomyelin Lipidosis

Drugs mentioned

• Olipudase alfa (olipudase alfa)

Snippet

Sponsor: Genzyme, a Sanofi Company | Conditions: Sphingomyelin Lipidosis | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2021-02-01