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Expert Rev Hematol. 2018 Feb;11(2):83-86. doi: 10.1080/17474086.2018.1423961. Epub 2018 Jan 6.

Measuring success in hemophilia gene therapy using a factor level & outcomes yardstick.

Author information

1
a Department of Medicine , University of Pittsburgh Medical Center , Pittsburgh , PA , USA.
2
b Hemophilia Center of Western Pennsylvania , Pittsburgh , PA , USA.

KEYWORDS:

Clotting factor; gene therapy; hemophilia; liver transplant; outcomes

PMID:
29300108
DOI:
10.1080/17474086.2018.1423961
[Indexed for MEDLINE]

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