Matches in SemOpenAlex for { <https://semopenalex.org/work/W4303193169> ?p ?o ?g. }
- W4303193169 endingPage "580" @default.
- W4303193169 startingPage "559" @default.
- W4303193169 abstract "Gene therapy for hemophilia using adeno-associated virus (AAV) derived vectors can reduce or eliminate patients' disease-related complications and improve their quality of life. Broad implementation globally will lead to societal gains and foster health equity. Several vector products each for factor IX (FIX) or factor VIII (FVIII) deficiency are in advanced clinical development. Safety data are reassuring. Efficacy data for up to 8 and 5 years, respectively, vary considerably among vector types and among individuals, but indicate significant reduction in bleeds and factor use. Products will soon be approved for marketing. This review highlights the relevant considerations for implementation of hemophilia gene therapy, specifically across a broad range of socioeconomic backgrounds globally, based on recent publications and our own experience. We address the current efficacy and safety data and relevant aspects of vector immunology. We then discuss pertinent implementation steps including pre-implementation and readiness assessments, considerations on cost, cost-effectiveness and payment models, approaches to education and informed consent, and the operational needs as well as the need for monitoring of health outcomes and implementation outcomes. To prevent a lag or complete lack of establishing access to this life-changing therapy option for all patients with hemophilia worldwide, adaptable pathways supported by collaborative and international efforts of all stakeholders are needed." @default.
- W4303193169 created "2022-10-07" @default.
- W4303193169 creator A5010364362 @default.
- W4303193169 creator A5039185570 @default.
- W4303193169 creator A5056195915 @default.
- W4303193169 creator A5063456712 @default.
- W4303193169 creator A5067542747 @default.
- W4303193169 creator A5073816998 @default.
- W4303193169 date "2022-10-01" @default.
- W4303193169 modified "2023-09-28" @default.
- W4303193169 title "Gene Therapy and Hemophilia: Where Do We Go from Here?" @default.
- W4303193169 cites W1969271703 @default.
- W4303193169 cites W1976329054 @default.
- W4303193169 cites W1988197108 @default.
- W4303193169 cites W2000118489 @default.
- W4303193169 cites W2004983645 @default.
- W4303193169 cites W2020155291 @default.
- W4303193169 cites W2047667011 @default.
- W4303193169 cites W2057767029 @default.
- W4303193169 cites W2065451039 @default.
- W4303193169 cites W2082025265 @default.
- W4303193169 cites W2095331703 @default.
- W4303193169 cites W2122304523 @default.
- W4303193169 cites W2130515212 @default.
- W4303193169 cites W2142301741 @default.
- W4303193169 cites W2151514148 @default.
- W4303193169 cites W2168114765 @default.
- W4303193169 cites W2170205772 @default.
- W4303193169 cites W2320744331 @default.
- W4303193169 cites W2398306036 @default.
- W4303193169 cites W2588392437 @default.
- W4303193169 cites W2741120766 @default.
- W4303193169 cites W2773253161 @default.
- W4303193169 cites W2773565340 @default.
- W4303193169 cites W2775538622 @default.
- W4303193169 cites W2883732986 @default.
- W4303193169 cites W2910456347 @default.
- W4303193169 cites W2944976798 @default.
- W4303193169 cites W2944993744 @default.
- W4303193169 cites W2951513309 @default.
- W4303193169 cites W2957860328 @default.
- W4303193169 cites W2979698216 @default.
- W4303193169 cites W2982694372 @default.
- W4303193169 cites W2989359658 @default.
- W4303193169 cites W2994542907 @default.
- W4303193169 cites W2996268542 @default.
- W4303193169 cites W2997125990 @default.
- W4303193169 cites W2998050720 @default.
- W4303193169 cites W3000219265 @default.
- W4303193169 cites W3003053451 @default.
- W4303193169 cites W3034062137 @default.
- W4303193169 cites W3035047148 @default.
- W4303193169 cites W3041796233 @default.
- W4303193169 cites W3047046471 @default.
- W4303193169 cites W3049590123 @default.
- W4303193169 cites W3049763201 @default.
- W4303193169 cites W3093323554 @default.
- W4303193169 cites W3095030514 @default.
- W4303193169 cites W3099555255 @default.
- W4303193169 cites W3100971054 @default.
- W4303193169 cites W3109958337 @default.
- W4303193169 cites W3110460373 @default.
- W4303193169 cites W3113310305 @default.
- W4303193169 cites W3120185015 @default.
- W4303193169 cites W3139253407 @default.
- W4303193169 cites W3146187792 @default.
- W4303193169 cites W3157904277 @default.
- W4303193169 cites W3158318636 @default.
- W4303193169 cites W3159651421 @default.
- W4303193169 cites W3171241404 @default.
- W4303193169 cites W3181927643 @default.
- W4303193169 cites W3186343535 @default.
- W4303193169 cites W3189383694 @default.
- W4303193169 cites W3200904070 @default.
- W4303193169 cites W3208678217 @default.
- W4303193169 cites W3210996225 @default.
- W4303193169 cites W3211832397 @default.
- W4303193169 cites W3213102199 @default.
- W4303193169 cites W3213236708 @default.
- W4303193169 cites W4205553739 @default.
- W4303193169 cites W4221076899 @default.
- W4303193169 cites W4223424509 @default.
- W4303193169 cites W4225571913 @default.
- W4303193169 cites W4229002408 @default.
- W4303193169 cites W4239823957 @default.
- W4303193169 cites W4281664094 @default.
- W4303193169 cites W4285992907 @default.
- W4303193169 doi "https://doi.org/10.2147/jbm.s371438" @default.
- W4303193169 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/36226233" @default.
- W4303193169 hasPublicationYear "2022" @default.
- W4303193169 type Work @default.
- W4303193169 citedByCount "3" @default.
- W4303193169 countsByYear W43031931692023 @default.
- W4303193169 crossrefType "journal-article" @default.
- W4303193169 hasAuthorship W4303193169A5010364362 @default.
- W4303193169 hasAuthorship W4303193169A5039185570 @default.
- W4303193169 hasAuthorship W4303193169A5056195915 @default.
- W4303193169 hasAuthorship W4303193169A5063456712 @default.