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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)

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: 01 Sep 2016

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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

    • 04 Jun 2015 According to a Genzyme Corporation media release, olipudase alfa has been granted Breakthrough Therapy designation by the U.S. FDA for the treatment of Niemann-Pick disease type B. The designation is supported by data from this trial.
    • 12 Feb 2015 According to a Genzyme media release, results were presented at the Lysosomal Disease Network's WORLD Symposium 2015.
    • 12 Feb 2015 Results were published in a Genzyme media release.
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