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Article

Gene therapy in hemophilia A: a cost-effectiveness analysis

24 Jul 2018

Abstract excerpt

Gene therapy provides a potential phenotypic cure for hemophilia, yet the cost of this novel treatment is high, tempering enthusiasm and raising questions regarding cost vs benefit. To evaluate the cost-effectiveness of gene therapy treatment of severe hemophilia A compared with prophylaxis with factor VIII (FVIII), we developed a Markov state-transition model to estimate the costs and effectiveness of severe...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 30042145
DOI: 10.1182/bloodadvances.2018021345

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