Search for an orphan drug

* (*) mandatory field

Other search option(s)

Orphan designation - USA

  • Substance: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene
  • ATC code : -
  • EU Number: -
  • Designation date : 30/04/2010
  • Sponsor of orphan designation : FONDAZIONE TELETHON


Treatment of Wiskott Aldrich syndrome.

Diseases list

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.