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An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)

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Trial Profile

An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 02 Jan 2024

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At a glance

  • Drugs Olipudase alfa (Primary)
  • Indications Niemann-Pick disease type B
  • Focus Adverse reactions
  • Sponsors Genzyme Corporation

    • Most Recent Events

    • 14 Nov 2023 Results assessing liver and lipid values at baseline and after 2years of olipudase alfa treatment in ongoing clinical trials and extensions (NCT02004691, NCT02292654 NCT02004704, NCT01722526) and Phase 1b trial presented at The Liver Meeting 2023: 74th Annual Meeting of the American Association for the Study of Liver Diseases
    • 09 Feb 2022 According to a Sanofi media release, These data were presented at the 18th annual WORLD Symposium held this week in San Diego, California.
    • 09 Feb 2022 Results published in the sanofi Media Release
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