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Orphan designation - Europe

  • Substance: Adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene
  • ATC code : -
  • EU Number: EU/3/11/917
  • Designation date : 27/10/2011
  • Sponsor of orphan designation : INSTITUT PASTEUR
  • Summary of Opinion


Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome).

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