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Orphan designation - Europe

  • Substance: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate aminotransferase gene
  • ATC code : -
  • EU Number: EU/3/12/974
  • Designation date : 21/03/2012
  • Sponsor of orphan designation : UNIQURE BIOPHARMA B.V.
  • Summary of Opinion


Treatment of primary hyperoxaluria type 1.

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