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Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive (CD34+) Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

Trial Profile

Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive (CD34+) Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

Status: Active, no longer recruiting
Phase of Trial: Phase I

Latest Information Update: 08 Dec 2021

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At a glance

  • Drugs AVR RD 01 (Primary)
  • Indications Fabry's disease
  • Focus Adverse reactions; First in man
  • Acronyms FACTs
  • Most Recent Events

    • 24 Nov 2021 Results published in an AVROBIO Media Release.
    • 24 Nov 2021 According to an AVROBIO media release, data presented at the 14th International Congress of Inborn Errors of Metabolism (ICIEM).
    • 04 Nov 2021 According to a AVROBIO media release, data from this trial will be presented at the 18th Annual WORLDSymposium in February 2022.
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