Search for an orphan drug

* (*) mandatory field

Other search option(s)

Orphan designation - Europe

  • Substance: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
  • ATC code : -
  • EU Number: EU/3/16/1716
  • Designation date : 29/08/2016
  • Sponsor of orphan designation : ADVANCED BIOTHERAPEUTICS CONSULTING SARL
  • Summary of Opinion


Treatment of Duchenne muscular dystrophy.

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.