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Novel approaches to hemophilia therapy: successes and challenges

Valder R. Arruda, Bhavya S. Doshi and Benjamin J. Samelson-Jones

Abstract

New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to non-factor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies. To date, none specifically address the challenge of dispersing treatment to the developing world.

  • Submitted August 1, 2017.
  • Accepted October 3, 2017.