{"id":948,"date":"2022-09-13T10:22:30","date_gmt":"2022-09-13T10:22:30","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/?page_id=948"},"modified":"2026-04-02T06:04:34","modified_gmt":"2026-04-02T06:04:34","slug":"disease-progression","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/disease-progression\/","title":{"rendered":"Disease Progression"},"content":{"rendered":"<div id=\"acf-block-6418c8d2969f9\" 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 can progress to serious complications regardless of phenotype<sup>1<\/sup><\/h1>\n            <\/div>\n<\/div>\n\n<div id=\"acf-block-6418c8d296a3f\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6418c8d296a59\" class=\"block-wysiwyg\">\n            <h2>GAG accumulation can lead to progressive organ damage<sup>2<\/sup><\/h2>\n<h3>Recognize the signs and symptoms of Morquio A, a multi-systemic genetic condition<sup>3,4<\/sup><\/h3>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6418c8d297837\" class=\"block split-content equal-bias content-align-top\" 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                                                                                                                                                                                                                                                                                    \n                                    \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image\">\n                                                                                            <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/10\/disease-progression-alt.png?v=0.47\" alt=\"morquio disease progression\" \/>                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\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>Neurological Complications\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Cervical myelopathy, cervical spinal instability, spinal cord compression<sup>3<\/sup><\/p>\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>Ear, Nose, and Throat Issues\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Conductive and sensorineural hearing loss, airway obstruction<sup>3<\/sup><\/p>\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>Gastrointestinal Issues\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Mild hepatosplenomegaly, hernias, loose stools, diarrhea, constipation, abdominal pain<sup>2,5,6<\/sup><\/p>\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>Musculoskeletal Issues\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                \n<p><strong>Classical phenotype<sup>5<\/sup><\/strong><\/p>\n<ul>\n<li class=\"p1\">Final adult height of &lt;120 cm (3 feet, 11 inches)<\/li>\n<li class=\"p1\">Overt spinal and skeletal\n<ul>\n<li class=\"p1\">Abnormalities<\/li>\n<li class=\"p1\">Joint laxity<\/li>\n<li class=\"p1\">Genu valgum<\/li>\n<li class=\"p1\">Abnormal gait<\/li>\n<li class=\"p1\">Pectus carinatum<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p><strong>Nonclassical phenotype<sup>5,7-9<\/sup><\/strong><\/p>\n<ul>\n<li>Final adult height of &gt;140 cm (4 feet, 7 inches)<\/li>\n<li>Hip subluxation or hip dysplasia, and\/or pain as the primary signs\/symptoms<\/li>\n<li>Less overt skeletal manifestations<\/li>\n<li>Symptoms often misdiagnosed as spondyloepiphyseal dysplasia and\/or bilateral Perthes disease<\/li>\n<li>Symptoms that may develop later in life<\/li>\n<\/ul>\n\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>Ophthalmological Issues\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Diffuse corneal clouding, cataracts, reduction in visual acuity<sup>3,10,11<\/sup><\/p>\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>Dental Issues\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Dentinogenesis imperfecta, hypodontia, pointed cusps, spade-shaped incisors, thin enamel, abnormal buccal surfaces<sup>12<\/sup><\/p>\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>Cardiac Issues\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Mitral and aortic valve stenosis and regurgitation, tricuspid regurgitation, hypertrophy<sup>3<\/sup><\/p>\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>Pulmonary Complications\n<\/h2>\n                                                            <\/div>\n                                                            <div class=\"accordion-content\">\n                                                                <p>Obstructive sleep apnea, respiratory infections, respiratory failure<sup>5,13<\/sup><\/p>\n                                                            <\/div>\n                                                        <\/div>\n                                                                                                                                                <\/div>\n                                        <\/div>\n                                    <\/div>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-6418c8d297897\" 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-6418c8d2978ba\" 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\/heterogeneity\/\" target=\"_self\">Learn more about how the heterogeneity of Morquio A results in different patients experiencing different symptoms<sup>7<\/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-6418c8d297901\" 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>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, Al-Jawad M, Berger KI, et al. Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA. <em>J Inherit Metab Dis<\/em>. 2013;36(2):309-322.\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>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>Hendriksz CJ, Berger KI, Giugliani R, et al. International guidelines for the management and treatment of Morquio A syndrome. <em>Am J Med Genet A<\/em>. 2015;167A(1):11-25.\n<\/span><\/li>\n                                                                                                                                                <li><span>Stepien KM, Bentley A, Chen C, et al. Non-cardiac manifestations in adult patients with mucopolysaccharidosis. <em>Front Cardiovasc Med<\/em>. 2022;9(839391).\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>Yeung AH, Cowan MJ, Horn B, Rosbe KW. Airway management in children with mucopolysaccharidoses. <em>Arch Otolaryngol Head Neck Surg<\/em>. 2009;135(1):73-79.\n<\/span><\/li>\n                                                                                                                                                <li><span>Lachman RS, Burton BK, Clarke LA, et al. Mucopolysaccharidosis IVA (Morquio A syndrome) and VI (Maroteaux-Lamy syndrome): under-recognized and challenging to diagnose. <em>Skeletal Radiol<\/em>. 2014;43(3):359-369. \n<\/span><\/li>\n                                                                                                                                                <li><span>D&#8217;Avanzo F, Zanetti A, De Filippis C, Tomanin R. Mucopolysaccharidosis Type VI, an Updated Overview of the Disease. <em>Int J Mol Sci<\/em>. 2021;22(24):13456.\n<\/span><\/li>\n                                                                                                                                                <li><span>Regier DS, Oetgen M, Tanpaiboon P. Mucopolysaccharidosis Type IVA. In: Adam MP, Bick S, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, eds. <em>GeneReviews<\/em><sup>\u00ae<\/sup>. Seattle (WA): University of Washington, Seattle; July 11, 2013.\n<\/span><\/li>\n                                                                                                                                                <li><span>Kinirons MJ, Nelson J. Dental findings in mucopolysaccharidosis type IV A (Morquio&#8217;s disease type A). <em>Oral Surg Oral Med Oral Pathol<\/em>. 1990;70(2):176-179.\n<\/span><\/li>\n                                                                                                                                                <li><span>Akyol MU, Alden TD, Amartino H, et al. Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. <em>Orphanet J Rare Dis<\/em>. 2019;14(1):137.\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-948","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 Disease Progression| VIMIZIM\u00ae (elosulfase alfa)<\/title>\n<meta name=\"description\" content=\"Review the signs, symptoms, and complications associated with the progression of Morquio A. 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