{"id":25,"date":"2024-11-20T17:12:51","date_gmt":"2024-11-20T17:12:51","guid":{"rendered":"https:\/\/www.vimizim.com\/en-us\/?page_id=25"},"modified":"2026-05-05T19:50:38","modified_gmt":"2026-05-05T19:50:38","slug":"key-facts","status":"publish","type":"page","link":"https:\/\/www.vimizim.com\/en-us\/key-facts\/","title":{"rendered":"Key Facts"},"content":{"rendered":"<div id=\"acf-block-674df741ed733\" class=\"page-header\">         \n            \n        <div class=\"header-image\" style=\"background-image: url(https:\/\/www.vimizim.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/12\/key_facts_hero_3.png?v=0.58)\"><\/div>\n     \n    <div class=\"header-content\">\n                    <h1>Get the details on Morquio A<\/h1>\n            <\/div>\n<\/div>\n\n<div id=\"acf-block-673f35dce2b0a\" class=\"block split-content equal-bias\" data-muted-autoplay=\"false\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t                                                                <div class=\"content-block first-block\">\n                                                                                                <h2>What is Morquio A?<\/h2>\n<p>Morquio A is a form of mucopolysaccharidosis (MPS), a type of lysosomal storage disorder. Also known as MPS IVA, Morquio A is a rare and progressive inherited disease that affects major organ systems in the body.<\/p>\n<h3>What causes Morquio A?<\/h3>\n<p>Enzymes are proteins that perform\u00a0 specific jobs in your body. People with Morquio A do not make enough of a specific enzyme, called\u00a0<i>N<\/i>-acetylgalactosamine-6-sulfatase, or\u00a0<strong>GALNS (gal\u2027en\u2027es)<\/strong>, which\u00a0breaks down and recycles cellular waste called\u00a0glycosaminoglycans (<strong>GAGs<\/strong>).<\/p>\n<p>When the body doesn&#8217;t produce enough of the enzyme, GAGs build up and can potentially cause serious physical problems in<\/p>\n<ul>\n<li>Tissues<\/li>\n<li>Bones<\/li>\n<li>Major organs<\/li>\n<\/ul>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\n                                                                                                                                                                                                                                                                                    \n                                    \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image\">\n                                                                                            <img decoding=\"async\" class=\"\" src=\"https:\/\/www.vimizim.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/11\/Cells-1.png?v=0.58\" alt=\"\" \/>                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-673f3a441aa6c\" class=\"block split-content equal-bias bg-band bg-band-white\" data-muted-autoplay=\"false\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t                                                                <div class=\"content-block first-block\">\n                                                                                                <h3>How do people inherit Morquio A?<\/h3>\n<p>Morquio A is a recessively inherited condition. This means that both parents must have a genetic mutation, or variant, in the\u00a0<em>GALNS<\/em>\u00a0gene to pass Morquio A on to their children.<\/p>\n<p>When 2 parents who are carriers have children, each pregnancy presents a 25% chance (1 in 4) of passing Morquio A on to that child.<\/p>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\n                                                                                                                                                                                                                                                                                    \n                                    \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image\">\n                                                                                            <img decoding=\"async\" class=\"\" src=\"https:\/\/www.vimizim.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/11\/106586_Inheretance_Graphic-1.png?v=0.58\" alt=\"\" \/>                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-673f3b153915f\" class=\"block boxed-content top-border\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\n<div id=\"acf-block-673f3b15394d2\" class=\"image-text-block content-align-center block-zero-bottom\">\n            <div class=\"image-block large\">\n            <figure>\n                <div class=\"image image-rounded\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/www.vimizim.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/12\/boxed_content_image_1.png?v=0.58\" alt=\"\" \/>                <\/div>\n            <\/figure>\n        <\/div>\n        <div class=\"content-block\">\n                            <p style=\"font-size: 1.2rem\">Since the symptoms of Morquio A may worsen over time, it&#8217;s important to look for early symptoms to ensure that you get timely management to reduce the impact of Morquio 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-673f3cf1beedd\" class=\"block split-content equal-bias\" data-muted-autoplay=\"false\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t                                                                <div class=\"content-block first-block\">\n                                                                                                <h2>What are the signs and symptoms of Morquio A?<\/h2>\n<p>Morquio A is<strong>\u00a0heterogeneous<\/strong>\u00a0(<strong>het\u00b7er\u00b7oh\u00b7gene\u00b7ee\u00b7ous<\/strong>), which means that it affects each person differently. The signs and symptoms you experience may be different from other people with the condition, and your Morquio A may progress at a different rate.<\/p>\n<p><strong>Individuals with Morquio A may have:<\/strong><\/p>\n                                                                                                                                                                                                        <div class=\"block accordion\">\n                                        <div class=\"wrapper\">\n                                            <div class=\"inner-wrapper\">\n                                                                                                                                                                                                                                                                            <div class=\"accordion-item\">\n                                                            <div class=\"accordion-title\">\n                                                                <div class=\"accordion-trigger\">\n                                                                    <span><\/span>\n                                                                    <span><\/span>\n                                                                <\/div>\n                                                                <h2>Skeletal Symptoms\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <ul>\n<li>Skeletal deformities<\/li>\n<li>Short trunk<\/li>\n<li>Weakness in the neck<\/li>\n<li>Spinal cord compression<\/li>\n<li>Knock-knees<\/li>\n<li>Overly flexible joints<\/li>\n<li>Abnormal walk<\/li>\n<li>Spinal problems<\/li>\n<li>Chest deformities<\/li>\n<\/ul>\n                                                            <\/div>\n                                                        <\/div>\n                                                                                                                                                                                                                            <div class=\"accordion-item\">\n                                                            <div class=\"accordion-title\">\n                                                                <div class=\"accordion-trigger\">\n                                                                    <span><\/span>\n                                                                    <span><\/span>\n                                                                <\/div>\n                                                                <h2>Nonskeletal Symptoms\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <ul>\n<li>Breathing problems<\/li>\n<li>Heart valve problems<\/li>\n<li>Muscle weakness<\/li>\n<li>Eye problems<\/li>\n<li>Hearing loss<\/li>\n<li>Dental problems<\/li>\n<li>Enlarged liver<\/li>\n<li>Enlarged spleen<\/li>\n<li>Limited energy<\/li>\n<\/ul>\n                                                            <\/div>\n                                                        <\/div>\n                                                                                                                                                <\/div>\n                                        <\/div>\n                                    <\/div>\n                                                                                                                                <p>It is important to know that Morquio A does not affect your ability to think and learn.<\/p>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\n                                                                                                                                                                                                                                                                                    \n                                    \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image\">\n                                                                                            <img decoding=\"async\" class=\"\" src=\"https:\/\/www.vimizim.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/12\/Houser_170303_2797_02-3.png?v=0.58\" alt=\"\" \/>                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-673f3cf1bf0f1\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-673f3cf1bf270\" class=\"block-wysiwyg\">\n            <h2>The symptoms of Morquio A progress over time<\/h2>\n<p>Morquio A is progressive, which means that symptoms get worse over time as GAGs build up in the body.<\/p>\n<p>For many people with Morquio A, symptoms can appear by age 2 or 3, while some show signs and symptoms at 6 months old. Others may not show initial symptoms (including hip stiffness due to hip subluxation and joint laxity, and pain) until the second decade of life.<\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"next\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-673f3dfc18895\" class=\"block-wysiwyg\">\n            <p style=\"text-align: right\"><a href=\"https:\/\/www.vimizim.com\/en-us\/impact-on-endurance\/\">Find out how Morquio A can impact endurance &gt;<\/a><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":3,"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-25","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Key Facts - BioMarin Vimizim Patient EN-US<\/title>\n<meta name=\"description\" content=\"Morquio A is a form of mucopolysaccharidosis (MPS), a type of lysosomal storage disorder. 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