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2018 ; 45
(2
): 92-96
Nephropedia Template TP
gab.com Text
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Twit Text #
English Wikipedia
The Potential Close Future of Hemophilia Treatment - Gene Therapy, TFPI
Inhibition, Antithrombin Silencing, and Mimicking Factor VIII with an Engineered
Antibody
#MMPMID29765291
Korte W
; Graf L
Transfus Med Hemother
2018[Apr]; 45
(2
): 92-96
PMID29765291
show ga
Summary Hemophilia is one of the best researched monogenic diseases. Hemophilia A
will affect approximately 1:5,000 male live births. In recent decades, great
progress has been made with the introduction of recombinant proteins in the 1990s
for therapy and prophylaxis, securing adequate availability and, with the
introduction of the prophylaxis concept, reducing the negative impact of
hemophilia on morbidity (especially arthropathy). Despite this progress, there
are still challenges to overcome to secure adequate prophylaxis and treatment:
for the time being, causal pharmacological hemophilia prophylaxis and therapy
requires repeated i.v. application on a regular basis. Although this approach
leads to a reduced comorbidity, it does not yet represent an optimized approach
with continuous reversal of the hemophilic defect, which would be the ideal
solution. This review summarizes the very new treatment strategies for the
treatment of hemophilia A and B.
free
free
free
