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Community register of orphan medicinal products


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Autologous CD34+ cells transduced with a lentiviral vector containing the human Wiskott-Aldrich syndrome gene

EU orphan designation number: EU/3/13/1196   
Active ingredient: Autologous CD34+ cells transduced with a lentiviral vector containing the human Wiskott-Aldrich syndrome gene
Indication: Treatment of Wiskott-Aldrich-syndrome
Sponsor: Généthon
1 bis rue de l’internationale, 91000 Evry, France

   Public summary of scientific opinion    

European Commission proceduresGoto top of the page

Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
14/10/2013 Orphan designation EMA/OD/104/13 (2013)6741 of 07/10/2013