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Blood. 2017 Nov 23;130(21):2251-2256. doi: 10.1182/blood-2017-08-742312. Epub 2017 Oct 10.

Novel approaches to hemophilia therapy: successes and challenges.

Author information

1
The Children's Hospital of Philadelphia, Philadelphia, PA.
2
Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA; and.
3
Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Philadelphia, PA.

Abstract

New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor 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.

PMID:
29018078
PMCID:
PMC5813735
DOI:
10.1182/blood-2017-08-742312
[Indexed for MEDLINE]
Free PMC Article

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