Navigating Speed Bumps on the Innovation Highway in Hemophilia Therapeutics.

ABSTRACT: The unprecedented emergence of novel therapeutics for both hemophilia A and B during the last half decade has been accompanied by the promise of even more extraordinary progress in ameliorative and curative strategies for both disorders. Paradoxically, the speed of innovation has created new dilemmas for persons with hemophilia and their physicians with respect to optimizing individual choices from the expanding menu of standard and novel therapies and approaches to symptom or risk reduction, and ultimately, to normalizing the hemophilia phenotype. Among the most disruptive new approaches, challenges remain in the form of the adverse reactions that have been observed with nonfactor therapies, as well as in the uncertain long-term safety profile of potentially curative gene therapy. Togethe

SUBMITTER: DiMichele DM 

PROVIDER: S-EPMC6407800 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature