{"id":25,"date":"2024-11-18T12:13:08","date_gmt":"2024-11-18T12:13:08","guid":{"rendered":"https:\/\/www.naglazyme.com\/en-us\/?page_id=25"},"modified":"2026-06-03T09:50:29","modified_gmt":"2026-06-03T09:50:29","slug":"about-mps-vi","status":"publish","type":"page","link":"https:\/\/www.naglazyme.com\/en-us\/about-mps-vi\/","title":{"rendered":"About MPS VI"},"content":{"rendered":"<div id=\"acf-block-69b29cffde5f8\" class=\"block wrapped-content block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-69b29cffde7a3\" class=\"block-wysiwyg\">\n            <h1>About MPS VI<\/h1>\n<h2>MPS VI (or Maroteaux\u2010Lamy syndrome) is a rare but serious genetic disease.<sup>1-3<\/sup><\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-674eeee229c22\" class=\"block boxed-content block-tight-top block-zero-bottom boxed-content-no-bg\">\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-674eeee22a002\" class=\"image-text-block content-reverse block-tight-top block-zero-bottom\">\n            <div class=\"image-block medium\">\n            <figure>\n                <div class=\"image image-rounded\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/www.naglazyme.com\/en-us\/wp-content\/uploads\/sites\/2\/2024\/11\/about-right-img.png?v=0.37\" alt=\"\" \/>                <\/div>\n            <\/figure>\n        <\/div>\n        <div class=\"content-block\">\n                            <h3>Only about 1100 people in the world are thought to have it.<sup>2<\/sup><\/h3>\n<p>People born with MPS VI tend to have a cluster of symptoms that may include<sup>3<\/sup>:<\/p>\n<ul>\n<li>Short stature<\/li>\n<li>Coarse facial features<\/li>\n<li>Stiff joints<\/li>\n<li>Breathing problems<\/li>\n<li>Difficulty walking\/hip pain<\/li>\n<\/ul>\n                    <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-673b5cbc84ca3\" class=\"block wrapped-content block-zero-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-673b5cbc84e39\" class=\"block-wysiwyg\">\n            <h2>A lysosomal storage disorder<\/h2>\n<p>The name \u201cMaroteaux-Lamy\u201d comes from the doctors who first described the disease in 1963. The other name of the disease, MPS VI, stands for the medical name of the disease, mucopolysaccharidosis VI.<sup>1<\/sup><\/p>\n<p>MPS VI is a kind of disease called a \u201clysosomal storage disorder\u201d or LSD. There are more than 40 known LSDs. In all of these diseases, the lack of specific enzymes causes waste to build up inside cells.<sup>1<\/sup><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-673b5cbc85cd4\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-673b5cbc85cfc\" class=\"block-wysiwyg\">\n            <h2>Inherited\u2014and often hard to diagnose<\/h2>\n<p>MPS VI is an inherited condition. A child who gets one MPS VI gene mutation or pathogenic variant from each parent will have the disease. There are different degrees of MPS VI. Some people have clear symptoms very early that grow worse quickly. Doctors call this \u201crapidly progressing\u201d MPS VI. Some people may not show signs until later and have symptoms that get worse more slowly. This is called \u201cslowly progressing\u201d MPS VI. Regardless of whether a person has \u201crapidly\u201d or \u201cslowly\u201d progressing MPS VI, the disease gets worse over time in all cases. This is why it is important to begin therapy as early as possible.<sup>3,4<\/sup><\/p>\n<h2>A treatment for MPS VI<\/h2>\n<p>NAGLAZYME<sup>\u00ae<\/sup> (galsulfase) replaces the natural enzyme that is missing in MPS VI. In timed tests, eligible people who took NAGLAZYME were able <a href=\"https:\/\/www.naglazyme.com\/en-us\/benefits-of-naglazyme\/\">to climb more stairs and walk farther than before<\/a>.<sup>5<\/sup><\/p>\n<p>In this section of NAGLAZYME.com, you can learn more about\u00a0<a href=\"https:\/\/www.naglazyme.com\/en-us\/what-happens-in-mps-vi\/\">MPS VI<\/a>, including how it\u00a0<a href=\"https:\/\/www.naglazyme.com\/en-us\/what-does-mps-vi-do-to-the-body\/\">affects the body<\/a>\u00a0and how it is\u00a0<a href=\"https:\/\/www.naglazyme.com\/en-us\/how-is-mps-vi-diagnosed\/\">diagnosed<\/a>.<\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-691f1c4da4222\" 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>Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, et al, eds. <em>The Metabolic and Molecular Bases of Inherited Disease.<\/em> 8th ed. New York, NY: McGraw-Hill; 2001:3421-3452.\n<\/span><\/li>\n                                                                                                                                                <li><span>Swiedler SJ, Beck M, Bajbouj M, et al. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). <em>Am J Med Genet A.<\/em> 2005;134A(2):144-151.\n<\/span><\/li>\n                                                                                                                                                <li><span>Clarke L, Ellaway C, Foster H. Understanding the early presentation of mucopolysaccharidosis disorders: results of a systematic literature review and physician survey. <em>J Inborn Errors Metab Screen.<\/em> 2018;6:1-12.\n<\/span><\/li>\n                                                                                                                                                <li><span>Akyol MU, Alden TD, Amartino H, et al. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. <em>Orphanet J Rare Dis.<\/em> 2019;14:118.\n<\/span><\/li>\n                                                                                                                                                <li><span>NAGLAZYME [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-673c5c6b61750\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-673c5c6b618b7\" class=\"block-wysiwyg\">\n            <p style=\"text-align: right;font-size: 1.1rem\"><a style=\"text-decoration: none\" href=\"https:\/\/www.naglazyme.com\/en-us\/what-happens-in-mps-vi\/\"><strong>NEXT PAGE &gt;<\/strong><\/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 v27.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>About MPS VI | Patient<\/title>\n<meta name=\"description\" content=\"MPS VI (or Maroteaux-Lamy syndrome) is a rare but serious genetic disease. 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