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Orphan designation - Europe
- Substance: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
- ATC code : -
- EU Number: EU/3/12/1091
- Designation date : 24/01/2013
- Sponsor of orphan designation : BLUEBIRD BIO FRANCE
- Summary of Opinion
Treatment of beta-thalassaemia intermedia and major.
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