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Orphan designation - Europe

  • Substance: Adeno-associated viral vector containing the human factor IX gene
  • ATC code : -
  • EU Number: EU/3/11/938
  • Designation date : 11/01/2012
  • Sponsor of orphan designation : UNIQURE BIOPHARMA B.V.
  • Summary of Opinion


Treatment of haemophilia B.

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