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Orphan designation - Europe

  • Substance: adeno-associated viral vector serotype 8 encoding the human ATP7B gene under the control of the human alpha-1 antitrypsin promoter
  • ATC code : -
  • EU Number: EU/3/15/1573
  • Designation date : 11/11/2015
  • Sponsor of orphan designation : ALIGEN THERAPEUTICS S.L.
  • Summary of Opinion


Treatment of Wilson's disease.

Diseases list

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