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Orphan designation - Europe

  • Substance: Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene
  • ATC code : -
  • EU Number: EU/3/18/2007
  • Designation date : 16/04/2018
  • Sponsor of orphan designation : INFORMATION NOT PROVIDED
  • Summary of Opinion


Treatment of glycogen storage disease type II (Pompe's disease).

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