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Orphan designation - Europe

  • Substance: Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene
  • ATC code : -
  • EU Number: EU/3/17/1849
  • Designation date : 20/03/2017
  • Sponsor of orphan designation : FREELINE THERAPEUTICS LTD
  • Summary of Opinion


Treatment of Fabry disease.

Diseases list

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