{"id":5368,"date":"2025-10-07T14:26:16","date_gmt":"2025-10-07T14:26:16","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=5368"},"modified":"2026-01-07T19:33:29","modified_gmt":"2026-01-07T19:33:29","slug":"hemophilia-publications","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/hemophilia-publications\/","title":{"rendered":"Hemophilia A Publications"},"content":{"rendered":"<div id=\"acf-block-68710d6459d61\" class=\"hero hero-no-overlay hero-large-first-para\">\n            <div class=\"hero-background-image hero-background-image-desktop\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-drop.png?v=1.20);\"><\/div>\n\t    <div class=\"hero-background-image hero-background-image-mobile\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-drop-Mobile.png?v=1.20);\"><\/div>\n    \t<div class=\"overlay\"><\/div>\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"hero-content\">\n\t\t\t    \t\t\t        <ul class=\"breadcrumb\">\n\t\t\t            \t\t\t                                                                        <li><a href=\"https:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/\">Hemophilia A<\/a><\/li>\n                            \t\t\t                                                                        <li>Hemophilia A Publications<\/li>\n                            \t\t\t            \t\t\t        <\/ul>\n\t\t\t    \t\t\t\t\t\t\t\t\t\t\t\t    <h1>Publications\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn more about Hemophilia A and BioMarin therapies<\/p>\n\t\t\t\t\t\t\t\t\n                <p class=\"inline-buttons\">\n                                                        <\/p>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n<div id=\"acf-block-68cbe6561f6c8\" class=\"block wrapped-content bg-band bg-band-primary block-zero-top block-zero-bottom block-remove-top-padding block-remove-bottom-padding\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68cbe6561f825\" class=\"block-wysiwyg\">\n            <h3 style=\"margin-top: 1rem\">Featured Publications:<\/h3>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68c997f24072a\" class=\"block cards cards-carousel block-tight-top\" data-muted-autoplay=\"false\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                                                <div class=\"owl-carousel cards-carousel-slides\">\n                                                                                                                                                                                                                                                                                                                                                                                                                    \n                                \n                                <div class=\"carousel-slide\">\n                                    <div class=\"card card-fixed-button\">\n                                                                                <div class=\"card-content\">\n                                                                                                                                        <h4>An extrapolation of clinical data\n<\/h4>\n                                                                                                                                                                                                                                                    <p>Estimated long-term durability of valoctocogene roxaparvovec treatment in male patients with severe hemophilia A: an extrapolation of clinical data<\/p>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                                                                                                                            <\/div>\n                                                            <div class=\"card-actions\">\n                                                        \n                                                        <p><a class=\"button button-external\" href=\"https:\/\/doi.org\/10.1007\/s12325-025-03368-4\" target=\"_blank\">Read Publication<\/a><\/p>\n                                                                                                                                                                                        <\/div>\n                                    <\/div>\n                                <\/div>\n                                                                                                                                                                                                                                                                                                                                                                                            \n                                \n                                <div class=\"carousel-slide\">\n                                    <div class=\"card card-fixed-button\">\n                                                                                <div class=\"card-content\">\n                                                                                                                                        <h4>A matching-adjusted indirect comparison of bleeding frequency\n<\/h4>\n                                                                                                                                                                                                                                                    <p>Comparative effectiveness of valoctocogene roxaparvovec and efanesoctocog alfa in the treatment of severe hemophilia A: a matching-adjusted indirect comparison of bleeding frequency<\/p>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                                                                                                                            <\/div>\n                                                            <div class=\"card-actions\">\n                                                        \n                                                        <p><a class=\"button button-external\" href=\"https:\/\/doi.org\/10.1007\/s12325-025-03339-9\" target=\"_blank\">Read publication<\/a><\/p>\n                                                                                                                                                                                        <\/div>\n                                    <\/div>\n                                <\/div>\n                                                                                                                                                                                                                                                                                                                                                                                            \n                                \n                                <div class=\"carousel-slide\">\n                                    <div class=\"card card-fixed-button\">\n                                                                                <div class=\"card-content\">\n                                                                                                                                        <h4>A plain language summary on bleeding comparison\n<\/h4>\n                                                                                                                                                                                                                                                    <p>A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis<\/p>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                                                                                                                            <\/div>\n                                                            <div class=\"card-actions\">\n                                                        \n                                                        <p><a class=\"button button-external\" href=\"https:\/\/doi.org\/10.1080\/17474086.2025.2467861\" target=\"_blank\">Read publication<\/a><\/p>\n                                                                                                                                                                                        <\/div>\n                                    <\/div>\n                                <\/div>\n                                                                                                                                                                                                                                                                                                                                                                                            \n                                \n                                <div class=\"carousel-slide\">\n                                    <div class=\"card card-fixed-button\">\n                                                                                <div class=\"card-content\">\n                                                                                                                                        <h4>4-year results of the GENEr8-1 clinical trial\n<\/h4>\n                                                                                                                                                                                                                                                    <p>Plain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A<\/p>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                                                                                                                            <\/div>\n                                                            <div class=\"card-actions\">\n                                                        \n                                                        <p><a class=\"button button-external\" href=\"https:\/\/doi.org\/10.1080\/23995270.2025.2522071\" target=\"_blank\">Read publication<\/a><\/p>\n                                                                                                                                                                                        <\/div>\n                                    <\/div>\n                                <\/div>\n                                                                                                                                                                                                                                                                                                                                                                                            \n                                \n                                <div class=\"carousel-slide\">\n                                    <div class=\"card card-fixed-button\">\n                                                                                <div class=\"card-content\">\n                                                                                                                                        <h4>Clinical practice guidance\n<\/h4>\n                                                                                                                                                                                                                                                    <p>Corticosteroid use to mitigate transaminitis associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance<\/p>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                                                                                                                            <\/div>\n                                                            <div class=\"card-actions\">\n                                                        \n                                                        <p><a class=\"button button-external\" href=\"https:\/\/doi.org\/10.1016\/j.jtha.2025.02.042\" target=\"_blank\">Read guidelines<\/a><\/p>\n                                                                                                                                                                                        <\/div>\n                                    <\/div>\n                                <\/div>\n                                                                        <\/div>\n                \t\t\t        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d55f2dd93bc\" class=\"block wrapped-content bg-band block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d55f2dd9518\" class=\"block-wysiwyg\">\n            <h3>Hemophilia A Publications List:<\/h3>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d0f9bc3a9c4\" class=\"block resources-listing\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\n            \t\t\t                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Publication<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/07\/Publications-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                <p><a class=\"button\" href=\"https:\/\/www.jthjournal.org\/article\/S1538-7836(25)00150-3\/fulltext\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Publications<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>Real World Evidence<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                                                                                                                                                            <p><a class=\"button\" href=\"https:\/\/www.jthjournal.org\/article\/S1538-7836(25)00150-3\/fulltext\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Publication<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/07\/Publications-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                <p><a class=\"button\" href=\"https:\/\/link.springer.com\/article\/10.1007\/s12325-025-03368-4\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Publications<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Estimated long-term durability of valoctocogene roxaparvovec treatment in male patients with severe hemophilia a: An extrapolation of clinical data\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                                                                                                                                                            <p><a class=\"button\" href=\"https:\/\/link.springer.com\/article\/10.1007\/s12325-025-03368-4\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Publication<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/07\/Publications-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                <p><a class=\"button\" href=\"https:\/\/link.springer.com\/article\/10.1007\/s12325-025-03339-9\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Publications<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Comparative effectiveness of valoctocogene roxaparvovec and efanesoctocog alfa in the treatment of severe hemophilia a: A matching-adjusted indirect comparison of bleeding frequency\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                                                                                                                                                            <p><a class=\"button\" href=\"https:\/\/link.springer.com\/article\/10.1007\/s12325-025-03339-9\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Plain Language Summaries<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/11\/Educational-Materials-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                <p><a class=\"button\" href=\"https:\/\/www.tandfonline.com\/doi\/abs\/10.1080\/23995270.2025.2522071\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Publications<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Plain language summary of 4-year results of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia a\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                                                                                                                                                            <p><a class=\"button\" href=\"https:\/\/www.tandfonline.com\/doi\/abs\/10.1080\/23995270.2025.2522071\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Plain Language Summaries<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/11\/Educational-Materials-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                <p><a class=\"button\" href=\"https:\/\/www.tandfonline.com\/doi\/abs\/10.1080\/17474086.2025.2467861\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Publications<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>A plain language summary on indirectly comparing bleeding after valoctocogene roxaparvovec gene therapy to bleeding with emicizumab prophylaxis\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                                                                                                                                                            <p><a class=\"button\" href=\"https:\/\/www.tandfonline.com\/doi\/abs\/10.1080\/17474086.2025.2467861\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Plain Language Summaries<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/11\/Educational-Materials-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                <p><a class=\"button\" href=\"https:\/\/www.tandfonline.com\/doi\/abs\/10.2217\/frd-2023-0007\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Plain Language Summaries<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Plain language summary of the GENEr8-1 clinical trial of valoctocogene roxaparvovec gene therapy for hemophilia A\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                                                                                                                                                            <p><a class=\"button\" href=\"https:\/\/www.tandfonline.com\/doi\/abs\/10.2217\/frd-2023-0007\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Selected Publications<\/p>\n                                                <div class=\"resource-split-layout\">\n                            <div class=\"resource-image-block\">\n                                <div class=\"resource-image\">\n                                                                            <div class=\"resource-feature-image\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/07\/Publications-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2023\/05\/Hemophilia-A-Selected-Publications-List_FEB-2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Publications<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Access selected clinical, pre-clinical and other publications for Hemophilia A\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                            \t                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2023\/05\/Hemophilia-A-Selected-Publications-List_FEB-2026.pdf?v=1.20\" target=\"_blank\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                            \t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":4701,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-5368","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Hemophilia A Publications | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"Learn more about Hemophilia A and BioMarin therapies\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/hemophilia-publications\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" 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