{"id":2,"date":"2025-07-07T13:19:12","date_gmt":"2025-07-07T13:19:12","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=2"},"modified":"2026-03-09T14:21:11","modified_gmt":"2026-03-09T14:21:11","slug":"home","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/","title":{"rendered":"Home"},"content":{"rendered":"<div id=\"acf-block-686bcaeba5ec9\" class=\"hero hero-blue hero-video\">\n                            <div class=\"background-video\">\n            <video class=\"background-video-desktop\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Homepage_desktop_adjusted.mp4?v=1.20\" loop autoplay=\"\" playsinline=\"\" muted=\"\" preload=\"\">\n            <\/video>\n            <video class=\"background-video-mobile\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/08\/Homepage_mobile.mp4?v=1.20\" loop autoplay=\"\" playsinline=\"\" muted=\"\" preload=\"\">\n            <\/video>\n        <\/div>\n                    <a class=\"video-control video-control-play\" href=\"#\">Play<\/a>\n            <a class=\"video-control video-control-pause\" href=\"#\">Pause<\/a>\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\t\t\t\t\t\t\t\t    <h1>BioMarin Medical\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>An online medical resource for healthcare providers in the United States to learn about our latest research, ongoing clinical trials, and the therapeutic areas where we focus<\/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-686e8c5db4527\" class=\"block wrapped-content block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-686e8c5db4630\" class=\"block-wysiwyg\">\n            <h2>Disease States and Programs<\/h2>\n<p>BioMarin has therapies either in development or approved by the US Food and Drug Administration (FDA) in the following disease areas:<\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-686e8c5db47e0\" class=\"block cards cards-2-col block-zero-top block-zero-bottom\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"cards-title\">\n\t\t\t    \t\t\t\t\t\t\t<\/div>\n\t\t\t<div class=\"cards-wrapper\">\n\t\t\t    \n<div id=\"acf-block-686e8c5db4df2\" class=\"card\" data-muted-autoplay=\"false\">\n    \t    \n\n                    <img decoding=\"async\" class=\"card-image\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/HP_Enzyme.jpg?v=1.20\" alt=\"DNA graphic\"\/>        \n\t    <div class=\"card-content\">\n                            <h4>Enzyme Therapies\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/phenylketonuria-pku\/\" target=\"_self\">About Phenylketonuria<\/a><\/p>\n                                                                                                                                                                                                                                                                        \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/mucopolysaccharidosis-mps\/\" target=\"_self\">About Mucopolysaccharidosis<\/a><\/p>\n                                                                                                                                                                                                                                                                        \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/neuronal-ceroid-lipofuscinosis-type-2-cln2\/\" target=\"_self\">About  Neuronal Ceroid Lipofuscinosis Type 2<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-686e8c5db4ccd\" class=\"card\" data-muted-autoplay=\"false\">\n    \t    \n\n                    <img decoding=\"async\" class=\"card-image\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/HP_Skeletal.jpg?v=1.20\" alt=\"Bones graphic\"\/>        \n\t    <div class=\"card-content\">\n                            <h4>Skeletal Conditions\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/achondroplasia\/\" target=\"_self\">About Achondroplasia<\/a><\/p>\n                                                                                                                                                                                                                                                                        \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/hypochondroplasia\/\" target=\"_self\">About Hypochondroplasia<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-68efa3c525e0f\" class=\"card\" data-muted-autoplay=\"false\">\n    \t    \n\n                    <img decoding=\"async\" class=\"card-image\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/HP_HemophiliaA.jpg?v=1.20\" alt=\"Red blood cells graphic\"\/>        \n\t    <div class=\"card-content\">\n                            <h4>Hemophilia A\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/\" target=\"_self\">About Hemophilia A<\/a><\/p>\n                                                                                                                                                                                                                                                                        \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/player.vimeo.com\/video\/1101350444\" target=\"_self\">About Gene Therapy<\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-686e8c5db4f8a\" class=\"card\" data-muted-autoplay=\"false\">\n    \t    \n\n                    <img decoding=\"async\" class=\"card-image\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/HP_Innovation.jpg?v=1.20\" alt=\"Cells graphic\"\/>        \n\t    <div class=\"card-content\">\n                            <h4>Innovation\n<\/h4>\n        \n                                                                                                                                                                                                                                                                                    \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/duchenne-muscular-dystrophy-dmd\/\" target=\"_self\">About Duchenne Muscular Dystrophy<\/a><\/p>\n                                                                                                                                                                                                                                                                        \n                                                        \n                            <p><a class=\"button button-text button-arrow\" href=\"https:\/\/medical.biomarin.com\/en-us\/enpp1\/\" target=\"_self\">About ENPP1 Deficiency <\/a><\/p>\n                                                                                    <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-68be9dcacc18a\" class=\"block row-block medical-pipeline bg-band bg-band-secondary block-zero-top block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                            <h2>Pipeline and Clinical Development Programs<\/h2>\n                        \n                            <p>At BioMarin, we push the boundaries of medical possibility to make a transformative impact. We are dedicated to improving the lives of individuals living with rare genetic disorders. We do so by investing in groundbreaking research and developing innovative treatments.<\/p>\n<p><strong>Learn more about BioMarin research and clinical trials with treatments that utilize the following therapeutic technologies:<\/strong><\/p>\n            \n                            <div class=\"pipeline-wrapper\">\n                                    <div class=\"pipeline-col\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/gene-therapy-logo.png?v=1.20\" alt=\"Icon of DNA\"\/>                        \n                                                    <p><span class=\"glossaryLink\"  aria-describedby=\"tt\"  data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Gene therapies&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Gene therapy aims to treat or correct genetic disorders by introducing or modifying genetic material within a person&amp;#039;s cells. This is achieved by transferring therapeutic genes into the body to replace faulty or missing genes, allowing cells to produce functional proteins and restore normal cellular function. &amp;lt;sup&amp;gt;5&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"  data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex='0' role='link'>Gene Therapies<\/span><\/p>\n                        \n                                            <\/div>\n                                    <div class=\"pipeline-col\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Small-molecule-logo.png?v=1.20\" alt=\"Icon of interconnected circles\"\/>                        \n                                                    <p><span class=\"glossaryLink\"  aria-describedby=\"tt\"  data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Small molecules&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Small molecule therapy refers to a type of medical treatment that utilizes small, chemically synthesized compounds to interact with specific proteins or enzymes in the body. These molecules can modulate cellular pathways, signaling, or biochemical processes. Small molecule therapy is a versatile approach used to target a wide range of diseases, including infectious and metabolic disorders.&amp;lt;sup&amp;gt;6,7&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"  data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex='0' role='link'>Small Molecules<\/span><\/p>\n                        \n                                            <\/div>\n                                    <div class=\"pipeline-col\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Antisense-Oligonucleotides-logo.png?v=1.20\" alt=\"Icon of muscle dimples\"\/>                        \n                                                    <p><span class=\"glossaryLink\"  aria-describedby=\"tt\"  data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Antisense oligonucleotides&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Antisense oligonucleotide therapy involves the use of short, single-stranded pieces of genetic material called oligonucleotides to selectively target and modify the splicing of RNA molecules within cells. By binding to specific RNA sequences, these oligonucleotides can influence gene expression. Antisense oligonucleotide therapy holds potential for treating genetic disorders and other conditions by modulating the production of disease-associated proteins.&amp;lt;sup&amp;gt;8,9&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"  data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex='0' role='link'>Antisense Oligonucleotides<\/span><\/p>\n                        \n                                            <\/div>\n                                    <div class=\"pipeline-col\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/07\/Antibodies-logo.png?v=1.20\" alt=\"Icon of an antibody\"\/>                        \n                                                    <p><span class=\"glossaryLink\"  aria-describedby=\"tt\"  data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;Biologics&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;&amp;lt;!-- wp:paragraph --&amp;gt;Biologic therapy involves the use of living organisms or their products to treat various medical conditions. These therapies often include proteins, biologics, or other complex molecules that are engineered to target specific disease processes in the body. Biologic therapies have revolutionized the treatment landscape, offering innovative approaches for conditions such as autoimmune diseases, cancer, and chronic inflammatory disorders.&amp;lt;sup&amp;gt;10,11&amp;lt;\/sup&amp;gt; (See references on the Pipeline and Clinical Development Programs page)&amp;lt;br\/&amp;gt;&amp;lt;!-- \/wp:paragraph --&amp;gt;&lt;\/div&gt;\"  data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex='0' role='link'>Biologics<\/span><\/p>\n                        \n                                            <\/div>\n                                <\/div>\n            \n                            <div class=\"pipeline-footer\">\n                    <a class=\"button button-arrow button-ghost\" href=\"https:\/\/medical.biomarin.com\/en-us\/pipeline-and-clinical-development-programs\/\" target=\"\">Explore the Pipeline and Clinical Development Programs Page<\/a>\n                <\/div>\n            \n        <\/div>\n    <\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-2","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>Home | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"An online medical resource for healthcare providers in the United States to learn about our latest research, ongoing clinical trials, and the therapeutic areas where we focus\" \/>\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\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" 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