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Orphan designation - Europe

  • Substance: Autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene
  • ATC code : -
  • EU Number: EU/3/09/623
  • Designation date : 29/04/2009
  • Sponsor of orphan designation : EGT SAN ROCCO ITALIA SRL
  • Summary of Opinion


Treatment of beta-thalassaemia intermedia and major.

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