Gene therapy with the Padua variant of a codon-optimised human factor IX gene etranacogene dezaparvovec in people with haemophilia B: Effects on patient-oriented outcomes measured using the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire in the HOPE-B study Conferences uri icon

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authors

  • Pipe, Steven
  • Abdelkader, Wael
  • Clearfield, Elizabeth
  • Kucher, Alexandra
  • Joseph, Bernard
  • Braverman, Julia
  • Galante, Nicholas
  • Monahan, Paul
  • Ibrahim, Quazi
  • Iorio, Alfonso
  • Germini, Federico
  • Skinner, Mark

publication date

  • April 2024