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Orphan designation - Europe

  • Substance: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
  • ATC code : -
  • EU Number: EU/3/14/1257
  • Designation date : 26/03/2014
  • Sponsor of orphan designation : STAAL
  • Summary of Opinion


Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency.

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