Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A
Sign Up to like & getrecommendations! Published in 2020 at "Journal of Medical Economics"
DOI: 10.1080/13696998.2020.1721508
Abstract: Abstract Aim: Hemophilia A is a genetic, chronic disorder classified by deficient or defective coagulation factor VIII (FVIII) that puts those affected at risk for spontaneous bleeding episodes, which lead to joint damage and chronic… read more here.
Keywords: treatment; gene therapy; therapy; hemophilia ... See more keywords
Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A
Sign Up to like & getrecommendations! Published in 2021 at "Haemophilia"
DOI: 10.1111/hae.14391
Abstract: Valoctocogene roxaparvovec is an investigational AAV5‐based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A. read more here.
Keywords: valoctocogene roxaparvovec; gene therapy; severe haemophilia;