x

Search for an orphan drug

* (*) mandatory field

Other search option(s)

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

Designation

Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency.

The documents contained in this web site are presented for information purposes only. The material is in no way intended to replace professional medical care by a qualified specialist and should not be used as a basis for diagnosis or treatment.