Do Patients Really Want These Therapies? Alleviating Patient Concerns to Bring Curative Therapies into the ‘Real World’

Time: 1:45 pm
day: Day Two


  • Understanding patient perspectives from the hemophilia, sickle cell and beta-thalassemia community in taking the gene therapy step
  • Why will some patients choose to wait to try gene therapy, even if they are eligible? Why will some be the first to “sign up”?
  • Identifying the best ways to engage patients after treatment has been administered: what is the follow-up protocol, for how long, and how to do that if the patient does not live near the center that administered the Gene Therapy
  • Bringing gene therapy into the public sphere