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1. Human gene therapy for hemophilia

Publication:

Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020

Subject(s):

Genetic Therapy
Hemophilia A -- drug therapy
Hemophilia B -- drug therapy
Clinical Trials as Topic
Drug Evaluation, Preclinical
Factor VIII -- therapeutic use
Factor IX -- therapeutic use
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

2. Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value : final report

Author(s):

Rind, David M., author
Walton, Surrey M., author
Agboola, Foluso, author
Herron-Smith, Serina, author
Quach, Danny, author
Chapman, Rick M., author
Pearson, Steven D., author
Bradt, Pamela, author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, November 20, 2020

Subject(s):

Antibodies, Monoclonal
Genetic Therapy
Hemophilia A -- drug therapy
United States

3. Emicizumab for hemophilia A with inhibitors: effectiveness and value : final evidence report

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, April 16, 2018

Subject(s):

Antibodies, Bispecific -- therapeutic use
Antibodies, Monoclonal -- therapeutic use
Hemophilia A -- drug therapy
United States