EU/3/14/1257

Table of contents

About

On 26 March 2014, orphan designation (EU/3/14/1257) was granted by the European Commission to Prof F.J.T. Staal, the Netherlands, for autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene for the treatment of recombination-activating gene 1 deficient severe combined immunodeficiency.

Key facts

Active substance
Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
Disease / condition
Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency
Date of decision
26/03/2014
Outcome
Positive
Orphan decision number
EU/3/14/1257

Sponsor's contact details

Prof. F.J.T. Staal
Leiden University Medical Center
Building 1, room L3-35
P.O. Box 9600
2300 RC Leiden
The Netherlands
Tel. +31 715 364 657
E-mail: f.j.t.staal@lumc.nl

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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