Novel approaches to hemophilia therapy: successes and challenges

Novel approaches to hemophilia therapy: successes and challenges

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 compli...

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Bibliographic Details
Published in:Blood Vol. 130; no. 21; pp. 2251 - 2256
Main Authors: Arruda, Valder R., Doshi, Bhavya S., Samelson-Jones, Benjamin J.
Format: Journal Article
Language:English
Published: United States Elsevier Inc 23-11-2017
American Society of Hematology
Subjects:
Animals
Genetic Therapy
Half-Life
Hemophilia A - genetics
Hemophilia A - therapy
Humans
Spotlight
Treatment Outcome
Online Access:Get full text
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Description
Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2017-08-742312