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Orphan designation - Europe

  • Substance: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
  • ATC code : -
  • EU Number: EU/3/12/1091
  • Designation date : 24/01/2013
  • Sponsor of orphan designation : BLUEBIRD BIO FRANCE
  • Summary of Opinion

Designation

Treatment of beta-thalassaemia intermedia and major.

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