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Gene therapy in hemophilia A: a cost-effectiveness analysis

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 fac...

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Bibliografiske detaljer
Udgivet i:Blood Adv
Main Authors: Machin, Nicoletta, Ragni, Margaret V., Smith, Kenneth J.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6058236/
https://ncbi.nlm.nih.gov/pubmed/30042145
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2018021345
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