{"id":5373,"date":"2025-10-07T14:45:34","date_gmt":"2025-10-07T14:45:34","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=5373"},"modified":"2026-06-05T20:54:44","modified_gmt":"2026-06-05T20:54:44","slug":"hemophilia-a-topics","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/hemophilia-a-topics\/","title":{"rendered":"Hemophilia A Topics"},"content":{"rendered":"<div id=\"acf-block-68da7583acb8e\" 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=1.27);\"><\/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=1.27);\"><\/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\t    <span class=\"section-title\"> As of May 29th, 2026, BioMarin has voluntarily withdrawn ROCTAVIAN (valoctocogene roxaparvovec-rvox) from the market<\/span>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t    <h1>Topics\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn about Hemophilia A topics<\/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-68da7583ad3f9\" class=\"block resources-listing\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\n                            <div class=\"listing-title\">\n                                            <h2>Real World Evidence\n<\/h2>\n                                                        <\/div>\n\t\t\t\t\t\t                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-article\">\n                                                    <p class=\"resource-type\">Featured<\/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\" data-format=\"Featured\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Real World Evidence\">Read publication<\/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>2025<\/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\" data-format=\"Featured\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Real World Evidence\">Read publication<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                            \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-68da7583ad221\" class=\"block resources-listing\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\n                            <div class=\"listing-title\">\n                                            <h2>Safety Profile and Efficacy\n<\/h2>\n                                                        <\/div>\n\t\t\t\t\t\t                                    \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\" data-format=\"Plain Language Summaries\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2024\" data-theme=\"Safety Profile and Efficacy\">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>2024<\/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\" data-format=\"Plain Language Summaries\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2024\" data-theme=\"Safety Profile and Efficacy\">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\">Featured<\/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\" data-format=\"Featured\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">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>2025<\/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\" data-format=\"Featured\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">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\" data-format=\"Publication\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">Read publication<\/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>2025<\/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\" data-format=\"Publication\" data-topic=\"Publications\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">Read publication<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-presentation\">\n                                                    <p class=\"resource-type\">Presentation<\/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\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/09\/BIC-2025_GENEr8-INH-pres_MED-BM-0017_SEPT_2025.pdf?v=1.27\" target=\"_blank\" data-format=\"Presentation\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"BIC\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>BIC<\/li>\n                                                                                                    <li>2025<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors: Results from a phase 1\/2 trial\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\/2025\/09\/BIC-2025_GENEr8-INH-pres_MED-BM-0017_SEPT_2025.pdf?v=1.27\" target=\"_blank\" data-format=\"Presentation\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"BIC\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/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\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/10\/HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00952-T1_HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00951-T2_EUCAN-ROC-00372.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"HEMO\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>HEMO<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                                    <li>Patient Reported Outcomes<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1\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\/2024\/10\/HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00952-T1_HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00951-T2_EUCAN-ROC-00372.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"HEMO\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/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\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/04\/WFH-2024-GENEr8-3-yr-HRQoL-poster_MMRC-BMN27-00862.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"WFH\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>WFH<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                                    <li>Patient Reported Outcomes<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENEr8-1\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\/2024\/04\/WFH-2024-GENEr8-3-yr-HRQoL-poster_MMRC-BMN27-00862.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"WFH\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">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\">Featured<\/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\" data-format=\"Featured\" data-topic=\"Plain Language Summaries\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">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                                                                                                    <li>2025<\/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\" data-format=\"Featured\" data-topic=\"Plain Language Summaries\" data-disease-state=\"Hemophilia A\" data-product=\"2025\" data-theme=\"Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/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\/2025\/05\/Congresses-1-1024x576.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\/12\/MMRC-BMN27-00758-ASH-2023-low-FVIII-Poster_approved-for-use.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"ASH\" data-product=\"2023\" data-theme=\"Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>ASH<\/li>\n                                                                                                    <li>2023<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Bleeding Outcomes in Participants With Factor VIII Activity &lt;5 IU\/dL Post-Gene Transfer in GENEr8-1\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\/12\/MMRC-BMN27-00758-ASH-2023-low-FVIII-Poster_approved-for-use.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"ASH\" data-product=\"2023\" data-theme=\"Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-presentation\">\n                                                    <p class=\"resource-type\">Presentation<\/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\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Hemostatic-results-for-up-to-6-years-following-treatment-with-valoctocogene-roxaparvovec-an-AAV5-hFVIII-SQ-gene-therapy.pdf?v=1.27\" target=\"_blank\" data-format=\"Presentation\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"ISTH\" data-product=\"2022\" data-theme=\"Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>ISTH<\/li>\n                                                                                                    <li>2022<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A\n<\/h2>\n                                                                                                <div class=\"resource-links\">\n                                                                                                                                                            \t                                   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class=\"inner-wrapper\">\n\n                            <div class=\"listing-title\">\n                                            <h2>Patient Reported Outcomes\n<\/h2>\n                                                        <\/div>\n\t\t\t\t\t\t                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/p>\n                                                <div class=\"resource-split-layout\">\n                 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href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/10\/HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00952-T1_HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00951-T2_EUCAN-ROC-00372.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"HEMO\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>HEMO<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                                    <li>Patient Reported Outcomes<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1\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\/2024\/10\/HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00952-T1_HEMO-2024-GENEr8-1_QoL-poster-MMRC-BMN27-00951-T2_EUCAN-ROC-00372.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"HEMO\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                    <\/div>\n                                                            <\/div>\n                        <\/div>\n                    <\/article>\n                                    \n                    \n                                                                                                                                                                                                                                                                                                                                \n                                                                                        \n                                        <article class=\"resource resource-poster\">\n                                                    <p class=\"resource-type\">Poster<\/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\/2025\/05\/Congresses-1-1024x576.png);\"><\/div>\n                                                                    <\/div>\n                                                                                                                                                                                                                                                                                                        <p><a class=\"button modal-pdfjs permitted\" href=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/04\/WFH-2024-GENEr8-3-yr-HRQoL-poster_MMRC-BMN27-00862.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"WFH\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">View content<\/a><\/p>\n                                                                                            <\/div>\n                            <div class=\"resource-information\">\n                                                                                                    <div class=\"resource-meta\">\n                                                                                    <ul class=\"tags\">\n                                                                                                    <li>Congresses<\/li>\n                                                                                                    <li>Hemophilia A<\/li>\n                                                                                                    <li>WFH<\/li>\n                                                                                                    <li>2024<\/li>\n                                                                                                    <li>Patient Reported Outcomes<\/li>\n                                                                                                    <li>Safety Profile and Efficacy<\/li>\n                                                                                            <\/ul>\n                                                                                                                    <\/div>\n                                                                                                                                    <h2>Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENEr8-1\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\/2024\/04\/WFH-2024-GENEr8-3-yr-HRQoL-poster_MMRC-BMN27-00862.pdf?v=1.27\" target=\"_blank\" data-format=\"Poster\" data-topic=\"Congresses\" data-disease-state=\"Hemophilia A\" data-region=\"WFH\" data-product=\"2024\" data-theme=\"Patient Reported Outcomes,Safety Profile and Efficacy\">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-5373","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Hemophilia A Topics | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"Learn about Hemophilia A topics\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link 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