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Orphan designation - Europe

  • Substance: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene
  • ATC code : -
  • EU Number: EU/3/12/998
  • Designation date : 06/06/2012
  • Sponsor of orphan designation : ORCHARD THERAPEUTICS (NETHERLANDS) B.V.
  • Summary of Opinion


Treatment of Wiskott-Aldrich syndrome.

Diseases list

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