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Orphan designation - Europe
- Substance: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
- ATC code : -
- EU Number: EU/3/14/1257
- Designation date : 26/03/2014
- Sponsor of orphan designation : STAAL
- Summary of Opinion
Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency.
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