{"id":222,"date":"2022-09-26T22:18:00","date_gmt":"2022-09-26T22:18:00","guid":{"rendered":"https:\/\/naglazymehcp-dev-001.azurewebsites.net\/en-us\/naglazyme\/?page_id=222"},"modified":"2023-05-25T15:05:45","modified_gmt":"2023-05-25T15:05:45","slug":"mps-vi-patient-care","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/naglazyme\/mps-vi-patient-care\/","title":{"rendered":"MPS VI patient care"},"content":{"rendered":"<div id=\"acf-block-6419cb1ebb3a2\" class=\"block wrapped-content block-tight-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6419cb1ebb431\" class=\"block-wysiwyg\">\n            <h1>MPS VI Patient Care<\/h1>\n<p>The multiple impacts of MPS VI require a combined strategy to manage specific symptoms while targeting underlying disease with enzyme replacement therapy (ERT). Early treatment may optimize outcomes and the potential benefits of ERT with NAGLAZYME<sup>\u00ae<\/sup> (galsulfase).<sup>1<\/sup><\/p>\n<p>Integrated care, usually supervised by a clinical geneticist, will involve multiple specialists to address specific needs.<sup>2<\/sup> Throughout treatment, regular multisystem monitoring and assessments are a must.<sup>2<\/sup> As patients age, it is important to plan the transition to adult care to ensure that the appropriate care is in place.<sup>3<\/sup><\/p>\n<h2>Integrated disease management<\/h2>\n<p>According to the 2019 management guidelines for mucopolysaccharidosis VI, integrated management of the progressive, multisystemic disease manifestations is essential.<sup>2<\/sup> Although treatment options have been limited in the past, enzyme replacement therapy with NAGLAZYME has demonstrated enduring benefits with long-term therapy and improved the outlook for patients with MPS VI.<sup>4<\/sup><\/p>\n<h2>Symptom-based care: it takes a team<\/h2>\n<p>While targeting the underlying disease, the coordinating clinician will also need to arrange for symptom-based care by specialists equipped to address the specific manifestations of the disease. Here are some key issues and recommended referrals<sup>5<\/sup>:<\/p>\n<ul>\n<li>Valvular heart disease: refer to a cardiologist<\/li>\n<li>Conductive and sensorineural hearing loss: refer to an audiologist for periodic hearing evaluations<\/li>\n<li>Corneal clouding and eventual significant visual impairment: refer to an ophthalmologist for initial and follow-up slit-lamp and fundoscopic exams<\/li>\n<li>Spinal cord compression secondary to thickening of the dura in the cervical canal (leads to myelopathy): refer to a neurosurgeon or orthopedist<\/li>\n<li>Airway obstruction, hyperventilation, and sleep apnea: refer to a pulmonologist for regular evaluation<\/li>\n<\/ul>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6419cb1ebb468\" 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\">Specific treatment for symptoms associated with MPS VI<sup>5-10<\/sup>\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 class=\"content-block\">\n                                <p><a class=\"button button-ghost button-download\" href=\"https:\/\/hcp.biomarin.com\/en-us\/naglazyme\/wp-content\/uploads\/sites\/2\/2023\/05\/Specific-treatment-for-symptoms-associated-with-MPS-VI-1.pdf?v=0.19\" target=\"_blank\">Download Now<\/a><\/p>\n                            <\/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-6419cb1ebb477\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6419cb1ebb49d\" class=\"block-wysiwyg\">\n            <h2>Symptom-based procedures<\/h2>\n<p>Symptom-based treatment addresses individual complications of MPS VI as they arise, and may include<sup>10<\/sup>:<\/p>\n<ul>\n<li>Antibiotics and other symptom-based medications<\/li>\n<li>Pain management<\/li>\n<li>Surgery<\/li>\n<li>Adaptive or supportive devices<\/li>\n<li>Physical or occupational therapy<\/li>\n<\/ul>\n<h2>The transition to adult care<sup>3<\/sup><\/h2>\n<p>As MPS VI patients age, it is important to plan the transition to adulthood, to ensure that the appropriate support is extended beyond the scope of pediatric care and parental supervision. This transition should be tailored to each patient\u2019s specific needs, so that those who can take over their own care have the tools they need, and those with limited capacities have the appropriate care and services in place to support them.<sup>3<\/sup><\/p>\n<h2>Planning required<sup>3<\/sup><\/h2>\n<p>The transition to adult care should include assessment of a patient\u2019s capacities to determine what he or she may be able to handle. Much of that consideration revolves around the patient\u2019s ability to communicate information about his or her condition. With that determination made, the right choices for continuing care should be offered. It is important to remember that this process is very gradual. The pediatric geneticist or other clinician coordinating the MPS VI patient\u2019s care may need to oversee the transition until the patient has achieved his or her capabilities and is integrated into the adult system. It is important to have a formal, site-specific transition strategy that includes joint visits with the pediatrician and adult patient physician. Families and patients should be encouraged to be involved in this process.<sup>11<\/sup><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6419cb1ebb4c6\" 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\">National Health Care Transition Center readiness assessment tool\n<\/h2>\n\t\t\t\t\t\t    \t\t\t\t\t\t    \t\t\t\t\t\t        <p>Download this questionnaire and share with patients and parents to plan for the transition to adult care. <\/p>\n\t\t\t\t\t\t    \t\t\t\t\t    <\/div>\n\t\t\t\t\t                                <div class=\"content-block\">\n                                <p><a class=\"button button-ghost button-download\" href=\"https:\/\/hcp.biomarin.com\/en-us\/naglazyme\/wp-content\/uploads\/sites\/2\/2022\/10\/GT-6CE-Leaving-Readiness-Assessment-Youth-F.pdf?v=0.3\" target=\"_blank\">Download Now<\/a><\/p>\n                            <\/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-6419cb1ebb4d3\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6419cb1ebb4e3\" class=\"block-wysiwyg\">\n            <p><a href=\"https:\/\/hcp.biomarin.com\/en-us\/naglazyme\/mps-vi-management\/\">Learn more about MPS VI disease management \u00bb<\/a><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6419cb1ebb8fd\" 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, Giugliani R, Schwartz I, <em>et al<\/em>. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. <em>J Pediatr<\/em>. 2006;148(4):533-539.\n<\/span><\/li>\n                                                                                                                                                <li><span>Akyol MU, Alden TD, Amartino H, <em>et al<\/em>. 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>Lampe C, McNelly B, Gevorkian AK, Hendriksz CJ, Lobzhanidze TV, P\u00e9rez-L\u00f3pez J, Stepien KM, Vashakmadze ND, Del Toro M. Transition of patients with mucopolysaccharidosis from paediatric to adult care. <em>Mol Genet Metab Rep<\/em>. 2019 Oct 21;21:100508. doi: 10.1016\/j.ymgmr.2019.100508. PMID: 31687335; PMCID: PMC6819742.\n<\/span><\/li>\n                                                                                                                                                <li><span>Giugliani R, Lampe C, Guffon N, <em>et al<\/em>. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux\u2013Lamy syndrome)\u201410-year follow-up of patients who previously participated in an MPS VI Survey Study. <em>Am J Med Genet A<\/em>. 2014;164A(8):1953-1964. doi:10.1002\/ajmg.a.36584.\n<\/span><\/li>\n                                                                                                                                                <li><span>Harmatz PR. Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Medscape website. https:\/\/emedicine.medscape.com\/article\/946474-overview. Updated March 20, 2017. Accessed January 2023.\n<\/span><\/li>\n                                                                                                                                                <li><span>NAGLAZYME [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2019. \n<\/span><\/li>\n                                                                                                                                                <li><span>Swiedler SJ, Beck M, Bajbouj M, <em>et al<\/em>. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). <em>Am J Med Genet A<\/em>. 2005;134A(2):144-150.\n<\/span><\/li>\n                                                                                                                                                <li><span>Lachman RS, Burton BK, Clarke LA. Mucopolysaccharidosis IVA (Morquio A syndrome) and VI (Maroteaux-Lamy syndrome): under-recognized and challenging to diagnose. <em>Skeletal Radiol<\/em>. 2014;43(3):359\u2013369. doi:10.1007\/s00256-013-1797-y.\n<\/span><\/li>\n                                                                                                                                                <li><span>Wraith JE. The mucopolysaccharidoses: a clinical review and guide to management. <em>Arch Dis Childhood<\/em>. 1995;72(3):263-267.\n<\/span><\/li>\n                                                                                                                                                <li><span>Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. <em>J Pediatr<\/em>. 2004;144(5 suppl):S3-S14.\n<\/span><\/li>\n                                                                                                                                                <li><span>American Academy of Pediatrics, American Academy of Family Physicians, American College of Physicians, Transitions Clinical Report Authoring Group, Cooley WC, Sagerman PJ. Supporting the health care transition from adolescence to adulthood in the medical home. <em>Pediatrics<\/em>. 2011;128(1):182-200. doi:10.1542\/peds.2011-0969.\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":4,"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-222","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>MPS VI patient care - BioMarin Naglazyme HCP EN-US<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/hcp.biomarin.com\/en-us\/naglazyme\/mps-vi-patient-care\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"MPS VI patient care - 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