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Adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene

  • INN (International Nonproprietary Name) : -
  • Code name : -
  • Chemical name or description : -
  • Type of product : Gene therapy product
  • Type of production : Biotechnology
  • Orphan designation : Europe
    • Orphan designation  - Europe

    • Diseases list
      • Severe hemophilia B
      • Moderately severe hemophilia B
      • Symptomatic form of hemophilia B in female carriers
    • Detailed information

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