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Orphan designation - Europe

  • Substance: adeno-associated virus vector encoding human phenylalanine hydroxylase
  • ATC code : -
  • EU Number: EU/3/19/2229
  • Designation date : 16/12/2019
  • Sponsor of orphan designation : BIOMARIN INTERNATIONAL LIMITED
  • Summary of Opinion


Treatment of phenylalanine hydroxylase deficiency.

Diseases list

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