{"id":946,"date":"2022-09-13T10:21:19","date_gmt":"2022-09-13T10:21:19","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/?page_id=946"},"modified":"2026-01-28T14:18:33","modified_gmt":"2026-01-28T14:18:33","slug":"mechanism-of-disease","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/mechanism-of-disease\/","title":{"rendered":"Mechanism of Disease"},"content":{"rendered":"<div id=\"acf-block-643e6e39d4e45\" class=\"page-header\">         \n            \n        <div class=\"header-image\" style=\"background-image: url(https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/09\/Nick2.png?v=0.47)\"><\/div>\n     \n    <div class=\"header-content\">\n                    <h1>Morquio A is caused by a deficiency of the GALNS enzyme<sup>1-3<\/sup><\/h1>\n            <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6e39d4eac\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-643e6e39d516b\" class=\"block-wysiwyg\">\n            <h2>Deficiency in enzyme activity results in glycosaminoglycan (GAG) accumulation leading to widespread cellular, tissue, and organ dysfunction<sup>4<\/sup><\/h2>\n<ul>\n<li>Deficient GALNS enzyme activity impairs lysosomal degradation of the GAGs keratan sulfate (KS) and chondroitin-6-sulfate (C6S)<sup>1,2,4<\/sup><\/li>\n<li>\n<p class=\"p1\">Accumulation of these GAGs in lysosomes leads to cellular dysfunction<sup>5<\/sup><\/p>\n<\/li>\n<\/ul>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6e39d5193\" class=\"block cards cards-2-col\">\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-643e6e39d51aa\" class=\"card\" data-muted-autoplay=\"false\">\n    \t    \n\n                    <img decoding=\"async\" class=\"card-image\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/11\/Cell1-441.png?v=0.47\" alt=\"\" \/>        \n\t    <div class=\"card-content\">\n                            <h4>Unaffected Cell\n<\/h4>\n        \n                                                                                                            <p>Unaffected cell without GAG accumulation in the lysosomes<sup>6<\/sup><\/p>\n                                                                                    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6e39d51bf\" class=\"card\" data-muted-autoplay=\"false\">\n    \t    \n\n                    <img decoding=\"async\" class=\"card-image\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/11\/Cell2-441.png?v=0.47\" alt=\"\" \/>        \n\t    <div class=\"card-content\">\n                            <h4>Morquio A Cell\n<\/h4>\n        \n                                                                                                            <p>Cell of a Morquio A patient with GAG accumulation in the lysosomes<sup>6<\/sup><\/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-643e6e39d51d3\" class=\"block boxed-content boxed-content-white\">\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-643e6e39d51ef\" class=\"image-text-block\">\n        <div class=\"content-block\">\n                                                    <p><a class=\"button button-text button-arrow\" href=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/disease-progression\/\" target=\"_self\">Learn more about how cellular dysfunction contributes to progressive organ damage<sup>4<\/sup><\/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-643e6e39d521d\" class=\"block references\">\n    <div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t    \t\t\t    <h4>References:\n<\/h4>\n\t\t\t\t\t\t                <ol>\n                                                                                                                        <li><span>Northover H, Cowie RA, Wraith JE. Mucopolysaccharidosis type IVA (Morquio syndrome): a clinical review. <em>J Inherit Metab Dis<\/em>. 1996;19(3):357-365.\n<\/span><\/li>\n                                                                                                                                                <li><span>Tomatsu S, Monta\u00f1o AM, Oikawa H, et al. Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment. <em>Curr Pharm Biotechnol<\/em>. 2011;12(6):931-945.\n<\/span><\/li>\n                                                                                                                                                <li><span>Harmatz PR, Mengel E, Geberhiwot T, et al. Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: an open-label, phase 2 study. <em>Am J Med Genet A<\/em>. 2017;173(2):375-383.\n<\/span><\/li>\n                                                                                                                                                <li><span>Harmatz P, Mengel KE, Giugliani R, et al. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. <em>Mol<\/em> <em>Genet Metab<\/em>. 2013;109(1):54-61.\n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Harmatz P, Beck M, et al. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA. <em>Mol Genet Metab<\/em>. 2013;110(1-2):54-64.\n<\/span><\/li>\n                                                                                                                                                <li><span>Bank RA, Groener JE, van Gemund JJ, et al. Deficiency in N-acetylgalactosamine-6-sulfate sulfatase results in collagen perturbations in cartilage of Morquio syndrome A patients. <em>Mol Genet Metab<\/em>. 2009;97(3):196-201.\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/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-946","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>Morquio A Mechanism of Disease |VIMIZIM\u00ae (elosulfase alfa)<\/title>\n<meta name=\"description\" content=\"Learn how a GALNS enzyme deficiency leads to the development of Morquio A. 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