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Orphan designation - Europe

  • Substance: Autologous CD34+ cells transduced with the W1.6_hWASP_WPRE lentiviral vector
  • ATC code : -
  • EU Number: EU/3/13/1196
  • Designation date : 07/10/2013
  • Sponsor of orphan designation : GENETHON
  • Summary of Opinion


Treatment of Wiskott-Aldrich syndrome.

Diseases list

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