{"id":950,"date":"2022-09-13T10:23:16","date_gmt":"2022-09-13T10:23:16","guid":{"rendered":"https:\/\/vimizimhcp-dev-001.azurewebsites.net\/en-us\/vimizim\/?page_id=950"},"modified":"2023-05-03T11:39:11","modified_gmt":"2023-05-03T11:39:11","slug":"heterogeneity","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/heterogeneity\/","title":{"rendered":"Heterogeneity"},"content":{"rendered":"<div id=\"acf-block-643e6f0606739\" 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=0.46)\"><\/div>\n     \n    <div class=\"header-content\">\n                    <h1>Morquio A is clinically heterogeneous<sup>1<\/sup><\/h1>\n            <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0606a65\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-643e6f0607f10\" class=\"block-wysiwyg\">\n            <h2>Over 200 variants of the <em>GALNS<\/em> gene give rise to wide genotypic and phenotypic heterogeneity<sup>2<\/sup><\/h2>\n<p>Regardless of the specific genotype or phenotype, Morquio A causes progressive end-organ damage that may result in life-threatening complications<sup>3<\/sup><\/p>\n<h3>Diverse presentations of Morquio A can complicate patient identification<sup>1<\/sup><\/h3>\n<ul>\n<li>Patients with a nonclassical phenotype may lack the overt skeletal manifestations that would lead to suspicion of Morquio A<sup>4<\/sup><\/li>\n<li>It is important to know all the signs and symptoms of Morquio A, especially when patients do not present with classic signs (e.g., bone deformity, short stature, and genu valgum)<sup>4<\/sup><\/li>\n<\/ul>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0607f2f\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<figure id=\"acf-block-643e6f0607f56\">\n    <div class=\"image image-rounded image-align-left\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/08\/sl2.png?v=0.46\" alt=\"\" \/>            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0607f5f\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-643e6f0607f6c\" class=\"block-wysiwyg\">\n            <h2>Identifying the wide spectrum of Morquio A presentations<\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0607f74\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<figure id=\"acf-block-643e6f0607f83\">\n    <div class=\"image image-responsive image-rounded image-align-left\">\n                    <div class=\"image-desktop\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/09\/man_vs_boy-1900x1046.jpg\" alt=\"\" \/>\t\t\t<\/div>\n\t\t\t<div class=\"image-mobile\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/08\/Patient-side-by-side.png?v=0.46\" alt=\"Morquio patients\" \/>\t\t\t<\/div>\n            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0607fa5\" class=\"block split-content equal-bias content-align-top bottom-border\" 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>Nonclassical musculoskeletal presentations can include:<sup>1,3<\/sup><\/h3>\n<ul>\n<li>Height frequently &gt;140 cm<\/li>\n<li>Hip stiffness and\/or pain as the primary presenting symptoms<\/li>\n<li>Presence of skeletal abnormalities, which are often not evident on examination<\/li>\n<\/ul>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\n                                                                                                <h3>Classical musculoskeletal presentations can include:<sup>1,3,5<\/sup><\/h3>\n<ul>\n<li>Height typically &lt;120 cm<\/li>\n<li>Presence of skeletal abnormalities, which are often evident on examination<\/li>\n<li>Chest abnormalities<\/li>\n<li>Genu valgum<\/li>\n<li>Presence of hypermobility<\/li>\n<\/ul>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0607fc5\" class=\"block call-to-action\">\n    <div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"box\" style=\"\">\n\t\t\t\t<div class=\"overlay\"><\/div>\n\t\t\t\t<div class=\"cta-content\">\n\t\t\t\t    \t\t\t\t\t<div class=\"cta-content-main\">\n                        <div class=\"content-block\">\n\t\t\t\t\t        \t\t\t\t\t        \t\t\t\t\t\t        <h2 class=\"h3\"><em>In an international registry, 1 out of 4 patients with Morquio A has a nonclassical phenotype.<sup>6<\/sup><\/em>\n<\/h2>\n\t\t\t\t\t\t    \t\t\t\t\t\t    \t\t\t\t\t    <\/div>\n\t\t\t\t\t                        <\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-643e6f0607fda\" 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-643e6f0608000\" class=\"image-text-block content-align-center\">\n        <div class=\"content-block\">\n                                                    <p><a class=\"button button-text button-arrow\" href=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/natural-history\/\" target=\"_self\">Learn more about the progressive nature of Morquio A<\/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-643e6f0608025\" 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>Akyol MU, Alden TD, Amartino H, <em><em>et al<\/em><\/em>. Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. <em>Orphanet J Rare Dis<\/em>. 2019;14:137. \n<\/span><\/li>\n                                                                                                                                                <li><span>Morrone A, Caciotti A, Atwood R, <em>et al<\/em>. Morquio A syndrome-associated mutations: a review of alterations in the <em>GALNS<\/em> gene and a new locus-specific database. <em>Hum Mutat<\/em>. 2014;35(11):1271-1279. doi: 10.1002\/humu.22635. \n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Harmatz P, Beck M, <em>et al<\/em>. 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>Bhattacharya K, Balasubramaniam S, Choy YS, <em>et al<\/em>. Overcoming the barriers to diagnosis of Morquio A syndrome. <em>Orphanet J Rare Dis<\/em>. 2014;9:192.\n<\/span><\/li>\n                                                                                                                                                <li><span>Tomatsu S, Monta\u00f1o AM, Oikawa H, <em>et al<\/em>. 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>Monta\u00f1o AM, Tomatsu S, Gottesman GS, Smith M, Orli T. International Morquio A registry: clinical manifestation and natural course of Morquio A disease. <em>J Inherit Metab Dis<\/em>. 2007;30(2):165-174.\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-950","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>Morquio A Heterogeneity |VIMIZIM\u00ae (elosulfase alfa<\/title>\n<meta name=\"description\" content=\"See how the clinical heterogeneity of Morquio A gives rise to diverse presentations that can complicate patient identification. 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