{"id":948,"date":"2022-09-13T10:22:30","date_gmt":"2022-09-13T10:22:30","guid":{"rendered":"https:\/\/vimizimhcp-dev-001.azurewebsites.net\/en-us\/vimizim\/?page_id=948"},"modified":"2023-05-13T10:37:05","modified_gmt":"2023-05-13T10:37:05","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=0.46)\"><\/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 disorder<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=0.46\" 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, 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>2<\/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>3,5<\/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>2,6,7<\/sup><\/strong><\/p>\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<p><strong>Nonclassical phenotype<sup>6,7<\/sup><\/strong><\/p>\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\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>8,9<\/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>10<\/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>3<\/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, <em>et al<\/em>. The Morquio A clinical assessment program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects.<em> Mol Genet Metab<\/em>. 2013;109(1):54-61. \n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Al-Jawad M, Berger KI, <em>et al<\/em>. 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, <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>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; Morquio families, International Morquio Organization (Carol Ann Foundation). Mucopolysaccharidosis type IVA: Morquio A syndrome. Slides presented at: 1st international Morquio family meeting. October 4, 2003; Broussard, LA. \n<\/span><\/li>\n                                                                                                                                                <li><span>Akyol MU, Alden TD, Amartino H, <em>et al<\/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>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>Fenzl CR, Teramoto K, Moshirfar M. Ocular manifestations and management recommendations of lysosomal storage disorders I: mucopolysaccharidoses. Clin Ophthalmol. 2015;9:1633-1644. \n<\/span><\/li>\n                                                                                                                                                <li><span>Leslie T, Siddiqui MAR, Aitken DA, Kirkness CM, Lee WR, Fern AI. Morquio syndrome: electron microscopy findings. Br J Opthalmol. 2005;89:917-929. \n<\/span><\/li>\n                                                                                                                                                <li><span>Kinirons MJ, Nelson J. Dental findings in mucopolysaccharidosis type IV A (Morquio&#8217;s disease type A). Oral Surg Oral Med Oral Pathol. 1990;70(2):176-179.\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 v27.4 - 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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