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Orphan designation withdrawn/expired - Europe

  • Substance: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel)
  • ATC code : -
  • EU Number: EU/3/12/1003
  • Designation date : 06/06/2012
  • Sponsor of orphan designation : BLUEBIRD BIO (NETHERLANDS) B.V.
  • Summary of Opinion


Treatment of adrenoleukodystrophy.

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