{"id":462,"date":"2022-08-10T14:19:11","date_gmt":"2022-08-10T18:19:11","guid":{"rendered":"https:\/\/campsiitestudy.com\/home\/"},"modified":"2023-06-09T09:59:45","modified_gmt":"2023-06-09T13:59:45","slug":"home","status":"publish","type":"page","link":"https:\/\/campsiitestudy.com\/pt-br\/","title":{"rendered":"Home"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"462\" class=\"elementor elementor-462 elementor-8\" data-elementor-post-type=\"page\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-40f7629 elementor-section-stretched hero-row elementor-section-full_width elementor-section-height-default elementor-section-height-default\" data-id=\"40f7629\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-no\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-a2ee3e4\" data-id=\"a2ee3e4\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-d6dc30d elementor-widget__width-auto elementor-absolute header-illustration cloud-1 elementor-widget elementor-widget-image\" data-id=\"d6dc30d\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"245\" height=\"94\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/cloud-1-1.svg\" class=\"attachment-full size-full wp-image-319\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-a5d0669 elementor-widget__width-auto elementor-absolute header-illustration cloud-2 elementor-widget elementor-widget-image\" data-id=\"a5d0669\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"212\" height=\"89\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/cloud-2.svg\" class=\"attachment-full size-full wp-image-292\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-8c13e9e elementor-widget__width-auto elementor-absolute header-illustration cloud-3 elementor-widget elementor-widget-image\" data-id=\"8c13e9e\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"245\" height=\"94\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/cloud-3.svg\" class=\"attachment-full size-full wp-image-295\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-43b39a0 elementor-widget__width-auto elementor-absolute header-illustration sun elementor-widget elementor-widget-image\" data-id=\"43b39a0\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_direction&quot;:&quot;negative&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"224\" height=\"224\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/sun.svg\" class=\"attachment-full size-full wp-image-301\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-4c3a7ed hero-inner-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"4c3a7ed\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-66 elementor-inner-column elementor-element elementor-element-db3c32b\" data-id=\"db3c32b\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-3cd8d7c elementor-widget elementor-widget-text-editor\" data-id=\"3cd8d7c\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h1>Um tratamento experimental \u00fanico para mucopolissacaridose tipo II (s\u00edndrome de Hunter)<\/h1>\n<p>A REGENXBIO, l\u00edder global em terapia g\u00eanica AAV, est\u00e1 atualmente inscrevendo participantes em um estudo cl\u00ednico de RGX-121, uma terapia g\u00eanica experimental \u00fanica para o tratamento potencial da mucopolissacaridose tipo II, tamb\u00e9m conhecida como MPS II ou s\u00edndrome de Hunter. O estudo cl\u00ednico, denominado Estudo CAMPSIITE\u2122, avaliar\u00e1 o efeito da RGX-121 em meninos com MPS II<sup>1<\/sup> neuronop\u00e1tica entre as idades de 4 meses e 5 anos. <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-c51415d cta-wrapper elementor-widget elementor-widget-text-editor\" data-id=\"c51415d\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p><a class=\"button\" href=\"#campsiite-study\">Saiba mais sobre o <span class=\"large-cta-text\">CAMPSIITE<\/span><\/a><\/p>\n<p><a class=\"text-link\" href=\"#contact-us\"><span class=\"large-cta-text\">Fale conosco<\/span><\/a><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-inner-column elementor-element elementor-element-eee5159\" data-id=\"eee5159\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap\">\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-39d4ca0 elementor-widget__width-auto elementor-absolute header-illustration tree-2 elementor-widget elementor-widget-image\" data-id=\"39d4ca0\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"300\" height=\"392\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-2.svg\" class=\"attachment-full size-full wp-image-304\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-4d4c8bb elementor-widget__width-auto elementor-absolute header-illustration tree-1 elementor-widget elementor-widget-image\" data-id=\"4d4c8bb\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"200\" height=\"261\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-1.svg\" class=\"attachment-full size-full wp-image-298\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-6d44d7b elementor-widget__width-auto elementor-absolute header-illustration tree-3 elementor-widget elementor-widget-image\" data-id=\"6d44d7b\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1.5,&quot;sizes&quot;:[]},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;],&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}}}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"200\" height=\"261\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-3.svg\" class=\"attachment-full size-full wp-image-307\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-6e4ee1a elementor-widget__width-auto elementor-absolute header-illustration tree-4 elementor-widget elementor-widget-image\" data-id=\"6e4ee1a\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;],&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}}}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"400\" height=\"523\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-4.svg\" class=\"attachment-full size-full wp-image-310\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-8fccfaf elementor-widget__width-auto elementor-absolute header-illustration tent elementor-widget elementor-widget-image\" data-id=\"8fccfaf\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1.5,&quot;sizes&quot;:[]},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;],&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}}}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"400\" height=\"235\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tent.svg\" class=\"attachment-full size-full wp-image-289\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-752a9f8 elementor-section-stretched light-green-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"752a9f8\" data-element_type=\"section\" data-e-type=\"section\" id=\"about-hunter-syndrome\" data-settings=\"{&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-833a9fc\" data-id=\"833a9fc\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-c9b777a elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"c9b777a\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h2>Sobre a S\u00edndrome de Hunter<\/h2>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-f550e32 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"f550e32\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>O que \u00e9 a Mucopolissacaridose Tipo II (MPS II)?<\/h3>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-0a5e48a elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"0a5e48a\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-871209e narrow-col\" data-id=\"871209e\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-c03adcc elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"c03adcc\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>A MPS II, ou s\u00edndrome de Hunter, \u00e9 uma condi\u00e7\u00e3o gen\u00e9tica rara que ocorre em aproximadamente um em cada 100.000 a 170.000 nascimentos masculinos.<sup>1<\/sup><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-56c449c elementor-invisible elementor-widget elementor-widget-image\" data-id=\"56c449c\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"463\" height=\"326\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/stat-pt-br-1.svg\" class=\"attachment-full size-full wp-image-566\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-487f125 callout-column elementor-invisible\" data-id=\"487f125\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;,&quot;animation_delay&quot;:150}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-856d904 elementor-widget elementor-widget-text-editor\" data-id=\"856d904\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<ul>\n<li>As pessoas que vivem com MPS II t\u00eam uma muta\u00e7\u00e3o no gene respons\u00e1vel pela produ\u00e7\u00e3o de uma enzima chamada iduronato-2-sulfatase (<em>IDS<\/em>)).<sup>1<\/sup><\/li>\n<li>As c\u00e9lulas precisam dessa enzima para decompor longas cadeias de mol\u00e9culas de a\u00e7\u00facar conhecidas como glicosaminoglicanos (GAGs).<sup>1<\/sup><\/li>\n<li>Se os GAGs se acumularem nas c\u00e9lulas do corpo, podem causar danos progressivos a muitos \u00f3rg\u00e3os, incluindo c\u00e9rebro, ouvidos, pulm\u00f5es, cora\u00e7\u00e3o, est\u00f4mago, intestinos, ossos, articula\u00e7\u00f5es, boca e pele. <sup>2<\/sup><\/li>\n<li>Os danos decorrentes do ac\u00famulo de GAG nas c\u00e9lulas s\u00e3o irrevers\u00edveis, destacando a import\u00e2ncia do diagn\u00f3stico e tratamento precoces. <sup>4<\/sup><\/li>\n<li>Os tratamentos atualmente aprovados para a s\u00edndrome de Hunter n\u00e3o abordam o impacto da MPS II no c\u00e9rebro. <sup>3<\/sup><\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-5755108 card card-row elementor-section-boxed elementor-section-height-default elementor-section-height-default elementor-invisible\" data-id=\"5755108\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-inner-column elementor-element elementor-element-348fe74\" data-id=\"348fe74\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-9feb85f elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"9feb85f\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>C\u00e9lula Saud\u00e1vel versus C\u00e9lula com MPS II<\/h3>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-b82149c screen-reader-text elementor-widget elementor-widget-text-editor\" data-id=\"b82149c\" data-element_type=\"widget\" data-e-type=\"widget\" id=\"overview-of-cell\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>C\u00c9LULA SAUD\u00c1VEL<\/h3>\n<ol>\n<li>Glicosaminoglicanos (GAGs): Mol\u00e9culas de a\u00e7\u00facar resultantes de fun\u00e7\u00f5es celulares normais<\/li>\n<li>O DNA normal codifica uma variedade de enzimas que decomp\u00f5em os GAGs<\/li>\n<\/ol>\n<p>Enzimas (essas enzimas est\u00e3o no lisossoma, n\u00e3o flutuando pelo citoplasma \u2013 portanto, Doen\u00e7as de Dep\u00f3sito Lisoss\u00f4mico)<\/p>\n<ol start=\"3\">\n<li>As enzimas decomp\u00f5em os GAGs<\/li>\n<\/ol>\n<h3>C\u00c9LULA COM MPS II NEURONOP\u00c1TICA<\/h3>\n<ol start=\"4\">\n<li>O DNA cont\u00e9m um c\u00f3digo defeituoso para a produ\u00e7\u00e3o de uma enzima espec\u00edfica que decomp\u00f5e os GAGs<\/li>\n<\/ol>\n<p>Citoplasma (no lisossoma, n\u00e3o no citoplasma)<\/p>\n<ol start=\"5\">\n<li>As enzimas s\u00f3 decomp\u00f5em parcialmente os GAGs, resultando em seu ac\u00famulo dentro da c\u00e9lula<\/li>\n<li> Em pacientes com MPS II neuronop\u00e1tica, o ac\u00famulo de GAG leva a danos nos tecidos e \u00f3rg\u00e3os em todo o corpo, inclusive neurodegenera\u00e7\u00e3o.<\/li>\n<\/ol>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-3db6687 elementor-invisible elementor-widget elementor-widget-image\" data-id=\"3db6687\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" aria-describedby=\"overview-of-cell\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese.jpg\" data-elementor-open-lightbox=\"yes\" data-elementor-lightbox-title=\"A Brazillian Portugese\" data-e-action-hash=\"#elementor-action%3Aaction%3Dlightbox%26settings%3DeyJpZCI6NTA3LCJ1cmwiOiJodHRwczpcL1wvY2FtcHNpaXRlc3R1ZHkuY29tXC93cC1jb250ZW50XC91cGxvYWRzXC8yMDIzXC8wNlwvQS1CcmF6aWxsaWFuLVBvcnR1Z2VzZS5qcGcifQ%3D%3D\">\n\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"2000\" height=\"959\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese.jpg\" class=\"attachment-full size-full wp-image-507\" alt=\"\" srcset=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese.jpg 2000w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese-300x144.jpg 300w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese-1024x491.jpg 1024w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese-768x368.jpg 768w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Brazillian-Portugese-1536x737.jpg 1536w\" sizes=\"(max-width: 2000px) 100vw, 2000px\" \/>\t\t\t\t\t\t\t\t<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-3b755cc elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"3b755cc\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Tipos de MPS II<\/h3>\n<p>Geralmente, h\u00e1 dois tipos de MPS II, que variam amplamente em termos de gravidade, taxa de progress\u00e3o e idade de in\u00edcio. <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-1c90de7 callout-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"1c90de7\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-7b9bfa3 card elementor-invisible\" data-id=\"7b9bfa3\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-fbdfcf9 elementor-widget elementor-widget-text-editor\" data-id=\"fbdfcf9\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h4>Atenuada<\/h4>\n<p>Na MPS II atenuada, a doen\u00e7a progride lentamente, com impacto m\u00ednimo ou nenhum impacto no c\u00e9rebro ou no desenvolvimento intelectual. <sup>1<\/sup><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-192b8b2 card\" data-id=\"192b8b2\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-78afd28 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"78afd28\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h4>Neuronop\u00e1tica<\/h4>\n<p>Na MPS II neuronop\u00e1tica, tamb\u00e9m conhecida como MPS II grave, h\u00e1 uma taxa mais r\u00e1pida de progress\u00e3o da doen\u00e7a e impacto significativo no c\u00e9rebro e no desenvolvimento intelectual. <sup>1<\/sup> A MPS II neuronop\u00e1tica \u00e9 respons\u00e1vel por at\u00e9 70%<sup>2<\/sup> das incid\u00eancias da doen\u00e7a. <sup>6<\/sup><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-759e5ec elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"759e5ec\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-66 elementor-inner-column elementor-element elementor-element-d8a829b elementor-invisible\" data-id=\"d8a829b\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-3dc3643 elementor-widget elementor-widget-text-editor\" data-id=\"3dc3643\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Na MPS II grave, ou na forma neuronop\u00e1tica, uma crian\u00e7a pode atingir os marcos iniciais do desenvolvimento, mas a taxa de aquisi\u00e7\u00e3o de habilidades diminui no in\u00edcio do desenvolvimento (geralmente 12 a 24 meses ap\u00f3s o nascimento). Esse atraso se torna mais aparente com o tempo, com habilidades de neurodesenvolvimento (aprendizagem e compreens\u00e3o, linguagem expressiva) e habilidades motoras finas movendo-se fora da faixa normal entre 24 e 29 meses. A aquisi\u00e7\u00e3o de habilidades geralmente se estabiliza por volta dos 5 a 6 anos, com a crian\u00e7a em um n\u00edvel de desenvolvimento semelhante a uma crian\u00e7a n\u00e3o afetada de 2 a 3 anos.<sup>1-5<\/sup>  <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-inner-column elementor-element elementor-element-c457825 elementor-invisible\" data-id=\"c457825\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;,&quot;animation_delay&quot;:150}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-c56a66d neuro-activities-ul elementor-widget elementor-widget-text-editor\" data-id=\"c56a66d\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>As atividades de neurodesenvolvimento incluem:<\/p><ul><li>Aprendizagem<\/li><li>Compreens\u00e3o<\/li><li>Linguagem expressiva<\/li><\/ul>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-9452835 elementor-widget-divider--view-line elementor-invisible elementor-widget elementor-widget-divider\" data-id=\"9452835\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeIn&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-ffebf0b symptoms elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"ffebf0b\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Sintomas<\/h3><p>Crian\u00e7as com MPS II podem apresentar uma variedade de sintomas, dependendo da idade de in\u00edcio e gravidade da condi\u00e7\u00e3o:<sup>1,2,6<\/sup><\/p><ul><li>Infec\u00e7\u00f5es de ouvido recorrentes<\/li><li>H\u00e9rnia umbilical ou inguinal (uma protuber\u00e2ncia de parte do intestino atrav\u00e9s da abertura na musculatura abdominal ou na regi\u00e3o da virilha)<\/li><li>Hidrocefalia (ac\u00famulo de l\u00edquido no c\u00e9rebro)<\/li><li>Engrossamento das narinas e l\u00e1bios<\/li><li>Macrocefalia (cabe\u00e7a maior que o normal)<\/li><li>Macroglossia ( l\u00edngua maior que o normal)<\/li><li>Hepatoesplenomegalia (ba\u00e7o e f\u00edgado aumentados)<\/li><li>Perda de audi\u00e7\u00e3o<\/li><li>Am\u00edgdalas aumentadas<\/li><li>Voz rouca<\/li><li>Dist\u00farbios do sono<\/li><li>Baixa estatura<\/li><li>Articula\u00e7\u00f5es r\u00edgidas e movimento limitado nas articula\u00e7\u00f5es<\/li><li>S\u00edndrome do t\u00fanel do carpo<\/li><li>Atraso no crescimento<\/li><li>Aparecimento tardio dos dentes ou espa\u00e7os amplos entre os dentes<\/li><li>Manchas esbranqui\u00e7adas na pele<\/li><li>Decl\u00ednio do neurodesenvolvimento, defici\u00eancia intelectual<\/li><li>Problemas comportamentais (hiperatividade, problemas de aten\u00e7\u00e3o, destrutividade, aus\u00eancia de medo)<\/li><\/ul>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-5573f1b elementor-widget-divider--view-line elementor-invisible elementor-widget elementor-widget-divider\" data-id=\"5573f1b\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeIn&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-833d0e3 additional-resources elementor-hidden-desktop elementor-hidden-tablet elementor-hidden-mobile elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"833d0e3\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Recursos adicionais<\/h3><p>Para mais informa\u00e7\u00f5es sobre a MPS II, visite os seguintes links:<\/p><ul><li><a href=\"https:\/\/mpssociety.org\/\" target=\"_blank\" rel=\"noopener\">National MPS Society (EUA)<\/a> (site dispon\u00edvel em ingl\u00eas)<\/li><li><a href=\"https:\/\/projectalive.org\/\" target=\"_blank\" rel=\"noopener\">Project Alive<\/a> (site dispon\u00edvel em ingl\u00eas)<\/li><li><a href=\"https:\/\/rarediseases.org\/rare-diseases\/mucopolysaccharidosis-type-ii-2\/\" target=\"_blank\" rel=\"noopener\">National Organization for Rare Disorders (NORD)<\/a> (site dispon\u00edvel em ingl\u00eas)<\/li><li><a href=\"https:\/\/casahunter.org.br\/home-en\/\" target=\"_blank\" rel=\"noopener\">Casa Hunter (Brasil)<\/a> (site dispon\u00edvel em ingl\u00eas e portugu\u00eas)<\/li><li><a href=\"https:\/\/www.mpssociety.ca\/\" target=\"_blank\" rel=\"noopener\">Canadian MPS Society for Mucopolysaccharide &amp; Related Diseases <\/a> (site dispon\u00edvel em ingl\u00eas)<\/li><li><a href=\"https:\/\/www.orpha.net\/consor\/cgi-bin\/Disease_Search.php?lng=EN&amp;data_id=131&amp;Disease_Disease_Search_diseaseGroup=mpsii&amp;Disease_Disease_Search_diseaseType=Pat&amp;Disease(s)\/group%20of%20diseases=Mucopolysaccharidosis-type-2&amp;title=Mucopolysaccharidosis%20type%202&amp;search=Disease_Search_Simple\" target=\"_blank\" rel=\"noopener\">Orphanet<\/a> (site dispon\u00edvel em tcheco, holand\u00eas, ingl\u00eas, franc\u00eas, alem\u00e3o, italiano, polon\u00eas, portugu\u00eas e espanhol)<\/li><\/ul>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-7001f8b elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"7001f8b\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Recursos adicionais<\/h3><p>Para mais informa\u00e7\u00f5es sobre a MPS II, visite os seguintes links:<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-1c3c54a elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"1c3c54a\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-no\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-e8aa059 elementor-invisible\" data-id=\"e8aa059\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-2ebc498 elementor-widget elementor-widget-text-editor\" data-id=\"2ebc498\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p><a href=\"https:\/\/mpssociety.org\/\" target=\"_blank\" rel=\"noopener\">National MPS Society (EUA)<\/a> (site dispon\u00edvel em ingl\u00eas)<\/p>\n<p><a href=\"https:\/\/projectalive.org\/\" target=\"_blank\" rel=\"noopener\">Project Alive<\/a> (site dispon\u00edvel em ingl\u00eas)<\/p>\n<p><a href=\"https:\/\/rarediseases.org\/rare-diseases\/mucopolysaccharidosis-type-ii-2\/\" target=\"_blank\" rel=\"noopener\">National Organization for Rare Disorders (NORD)<\/a> (site dispon\u00edvel em ingl\u00eas)<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-017f38e elementor-invisible\" data-id=\"017f38e\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-e4b74de elementor-widget elementor-widget-text-editor\" data-id=\"e4b74de\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p><a href=\"https:\/\/casahunter.org.br\/home-en\/\" target=\"_blank\" rel=\"noopener\">Casa Hunter (Brasil)<\/a> (site dispon\u00edvel em ingl\u00eas e portugu\u00eas)<\/p>\n<p><a href=\"https:\/\/www.mpssociety.ca\/\" target=\"_blank\" rel=\"noopener\">Canadian MPS Society for Mucopolysaccharide &amp; Related Diseases <\/a> (site dispon\u00edvel em ingl\u00eas)<\/p>\n<p><a href=\"https:\/\/www.orpha.net\/consor\/cgi-bin\/Disease_Search.php?lng=EN&amp;data_id=131&amp;Disease_Disease_Search_diseaseGroup=mpsii&amp;Disease_Disease_Search_diseaseType=Pat&amp;Disease(s)\/group%20of%20diseases=Mucopolysaccharidosis-type-2&amp;title=Mucopolysaccharidosis%20type%202&amp;search=Disease_Search_Simple\" target=\"_blank\" rel=\"noopener\">Orphanet<\/a> (site dispon\u00edvel em tcheco, holand\u00eas, ingl\u00eas, franc\u00eas, alem\u00e3o, italiano, polon\u00eas, portugu\u00eas e espanhol)<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-e679602 references elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"e679602\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Refer\u00eancias:<\/h3>\n<ol>\n<li>Rare Disease Database: Mucopolysaccharidosis type II. NORD \u2013 National Organization for Rare Disorders, Inc.; 2021. <a href=\"https:\/\/rarediseases.org\/rare-diseases\/mucopolysaccharidosis-type-ii-2\/\">https:\/\/rarediseases.org\/rare-diseases\/mucopolysaccharidosis-type-ii-2\/<\/a>.<\/li>\n<li>Escolar ML, Jones SA, Shapiro EG, Horovitz DDG, Lampe C, Amartino H. Practical management of behavioral problems in mucopolysaccharidoses disorders. <em>Mol Genet Metab. <\/em>2017;122:35-40.<\/li>\n<li>D\u2019Avanzo F, Rigon L, Zanetti A, Tomanin R. Mucopolysaccharidosis type II: one hundred years of research, diagnosis, and treatment. <em>Int J Mol Sci. <\/em>2020;21:1258.<\/li>\n<li>Muenzer J, Bodamer O, Burton B, et al. The role of enzyme replacement therapy in severe Hunter syndrome \u2013 an expert panel consensus. <em>Eur J Pediatr. <\/em>2012;171:181-188.<\/li>\n<li>Escolar M, Phillips D, Cho Y, Mulatya C, Nevoret M-L, Poe M. Natural history of neurodevelopment in neuronopathic mucopolysaccharidosis type II (MPS II): Mullen Scales of Early Learning (MSEL) visual reception, expressive and receptive language and fine motor scale developmental trajectories. Presented at 2022 annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Freiburg, Germany, August 2022. Abstract SSIEM22-2814.<\/li>\n<li>Scarpa M. Mucopolysaccharidosis type II. GeneReviews\u00ae [Internet]; 2018. <a href=\"https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK1274\/\">https:\/\/www.ncbi.nlm.nih.gov\/books\/NBK1274\/<\/a>. <\/li>\n<\/ol>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-a8b0ee9 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"a8b0ee9\" data-element_type=\"section\" data-e-type=\"section\" id=\"about-rgx-121-gene-therapy\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-e502aab\" data-id=\"e502aab\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-4bbae4d elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"4bbae4d\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h2>Sobre a Terapia G\u00eanica RGX-121<\/h2>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-505869f elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"505869f\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>O que s\u00e3o genes e terapia g\u00eanica?<sup>1-3<\/sup><\/h3>\n<p>Cada c\u00e9lula do corpo humano cont\u00e9m cromossomas compostos de genes. Esses genes consistem em DNA, que fornece instru\u00e7\u00f5es que dizem ao corpo como produzir prote\u00ednas, que s\u00e3o um elemento essencial para nossos organismos. O corpo humano cont\u00e9m milhares de genes.  <\/p>\n<p>A terapia g\u00eanica \u00e9 um tratamento que envolve a introdu\u00e7\u00e3o, remo\u00e7\u00e3o ou altera\u00e7\u00e3o do material gen\u00e9tico nas c\u00e9lulas de uma pessoa. Existem v\u00e1rias abordagens para a terapia g\u00eanica: <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-43ac9e3 callout-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"43ac9e3\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-inner-column elementor-element elementor-element-0088591 card orange-outline elementor-invisible\" data-id=\"0088591\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-bfc9cc1 elementor-widget elementor-widget-text-editor\" data-id=\"bfc9cc1\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h4>Adi\u00e7\u00e3o de genes<\/h4>\n<p>O fornecimento de um gene a uma pessoa, permitindo que o corpo dessa pessoa produza uma prote\u00edna funcional (\u00e0s vezes tamb\u00e9m chamada de substitui\u00e7\u00e3o de gene). A RGX-121 usa essa abordagem. <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-inner-column elementor-element elementor-element-8c25e4a card elementor-invisible\" data-id=\"8c25e4a\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;,&quot;animation_delay&quot;:150}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-6f91ba6 elementor-widget elementor-widget-text-editor\" data-id=\"6f91ba6\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h4>Edi\u00e7\u00e3o de genes<\/h4>\n<p>A inser\u00e7\u00e3o, substitui\u00e7\u00e3o ou exclus\u00e3o de DNA defeituoso dentro de um gene. <sup>2-3<\/sup><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-inner-column elementor-element elementor-element-0b5df88 card elementor-invisible\" data-id=\"0b5df88\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;,&quot;animation_delay&quot;:300}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-dfcf760 elementor-widget elementor-widget-text-editor\" data-id=\"dfcf760\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h4>Modifica\u00e7\u00e3o de RNA<\/h4>\n<p>A modifica\u00e7\u00e3o do RNA (uma mol\u00e9cula semelhante ao DNA que ajuda a c\u00e9lula a criar prote\u00ednas a partir de genes) para bloquear a produ\u00e7\u00e3o de prote\u00edna disfuncional de um gene mutante.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-c09acb4 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"c09acb4\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>O que \u00e9 a Tecnologia NAV<sup>\u00ae<\/sup>?<\/h3>\n<p>As terapias g\u00eanicas da REGENXBIO est\u00e3o sendo investigadas em estudos cl\u00ednicos e foram projetadas para fornecer genes funcionais a c\u00e9lulas usando vetores de v\u00edrus adenoassociados (adeno-associated virus &#8211; AAV) como \u201cve\u00edculos\u201d de entrega. Os vetores AAV s\u00e3o v\u00edrus modificados que n\u00e3o podem aumentar seus n\u00fameros ou se reproduzir e n\u00e3o s\u00e3o conhecidos por causar doen\u00e7as em humanos. <\/p>\n<p>Todas as nossas terapias g\u00eanicas investigativas usam vetores virais de nossa plataforma propriet\u00e1ria de fornecimento de genes, que chamamos de plataforma tecnol\u00f3gica NAV<sup>\u00ae<\/sup>. Milhares de pacientes receberam terapias g\u00eanicas AAV derivadas da nossa plataforma tecnol\u00f3gica NAV. <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-178efa4 elementor-invisible elementor-widget elementor-widget-video\" data-id=\"178efa4\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;video_type&quot;:&quot;vimeo&quot;,&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"video.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-wrapper elementor-open-inline\">\n\t\t\t<iframe class=\"elementor-video-iframe\" allowfullscreen allow=\"clipboard-write\" title=\"Reprodutor de v\u00eddeo vimeo\" src=\"https:\/\/player.vimeo.com\/video\/152453737?color&amp;autopause=0&amp;loop=0&amp;muted=0&amp;title=1&amp;portrait=1&amp;byline=1#t=\"><\/iframe>\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-114e336 elementor-widget-divider--view-line elementor-invisible elementor-widget elementor-widget-divider\" data-id=\"114e336\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeIn&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-4fa8fc4 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"4fa8fc4\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-b67c630 narrow-col elementor-invisible\" data-id=\"b67c630\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-b488f29 elementor-widget elementor-widget-text-editor\" data-id=\"b488f29\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>O que \u00e9 RGX-121?<\/h3>\n<p>A RGX-121 \u00e9 uma terapia g\u00eanica experimental \u00fanica para o tratamento potencial da MPS II. Foi projetada para usar o vetor NAV<sup>\u00ae<\/sup> AAV9 para fornecer o gene humano da iduronato-2-sulfatase (<em>IDS<\/em>) diretamente ao fluido que envolve o c\u00e9rebro, conhecido como l\u00edquido cefalorraquidiano (LCR). <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-3090435 callout-column elementor-invisible\" data-id=\"3090435\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-e048b5f elementor-widget elementor-widget-text-editor\" data-id=\"e048b5f\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<ul>\n<li>Depois que o vetor NAV<sup>\u00ae<\/sup> AAV9 fornece o gene IDS \u00e0s c\u00e9lulas no c\u00e9rebro, <strong>as c\u00e9lulas podem come\u00e7ar a produzir a enzima I2S necess\u00e1ria<\/strong> e podem fornecer uma fonte permanente de I2S no sistema nervoso central (SNC), que consiste no c\u00e9rebro e na medula espinhal.<\/li>\n<li>A produ\u00e7\u00e3o cont\u00ednua da enzima <strong>pode prevenir o decl\u00ednio do neurodesenvolvimento<\/strong> em crian\u00e7as que recebem a terapia g\u00eanica no in\u00edcio da vida e pode impedir que o dano piore em crian\u00e7as que recebem a terapia g\u00eanica em uma idade mais avan\u00e7ada.<\/li>\n<li>Ao contr\u00e1rio dos tratamentos atualmente dispon\u00edveis para MPS II, a RGX-121 pode atravessar a barreira hematoencef\u00e1lica (BHE), uma rede protetora de vasos sangu\u00edneos e c\u00e9lulas que separa o SNC do resto do corpo. Ao cruzar a BHE do SNC para atingir o resto do corpo, a RGX-121 <strong>pode abordar os efeitos sist\u00eamicos<\/strong> (aqueles que afetam o corpo como um todo) da MPS II.<sup>4<\/sup> <\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-31a73ac card card-row elementor-section-boxed elementor-section-height-default elementor-section-height-default elementor-invisible\" data-id=\"31a73ac\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-inner-column elementor-element elementor-element-1092c6a\" data-id=\"1092c6a\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-2340660 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"2340660\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Como a RGX-121 deve funcionar<\/h3>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-99df42d elementor-invisible elementor-widget elementor-widget-image\" data-id=\"99df42d\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" aria-describedby=\"transporter\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<a href=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese.jpg\" data-elementor-open-lightbox=\"yes\" data-elementor-lightbox-title=\"B Brazillian Portugese\" data-e-action-hash=\"#elementor-action%3Aaction%3Dlightbox%26settings%3DeyJpZCI6NTExLCJ1cmwiOiJodHRwczpcL1wvY2FtcHNpaXRlc3R1ZHkuY29tXC93cC1jb250ZW50XC91cGxvYWRzXC8yMDIzXC8wNlwvQi1CcmF6aWxsaWFuLVBvcnR1Z2VzZS5qcGcifQ%3D%3D\">\n\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"2000\" height=\"1096\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese.jpg\" class=\"attachment-full size-full wp-image-511\" alt=\"\" srcset=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese.jpg 2000w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese-300x164.jpg 300w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese-1024x561.jpg 1024w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese-768x421.jpg 768w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Brazillian-Portugese-1536x842.jpg 1536w\" sizes=\"(max-width: 2000px) 100vw, 2000px\" \/>\t\t\t\t\t\t\t\t<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-04a024e screen-reader-text elementor-widget elementor-widget-text-editor\" data-id=\"04a024e\" data-element_type=\"widget\" data-e-type=\"widget\" id=\"transporter\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Transportador (AAV)<\/h3>\n<ol>\n<li>O DNA viral \u00e9 removido<\/li>\n<li>O gene <em>IDS<\/em> humano funcional \u00e9 inserido<\/li>\n<li>O tratamento \u00e9 administrado no sistema nervoso central, guiado por tomografia computadorizada, enquanto o paciente est\u00e1 dormindo<\/li>\n<li>O vetor une-se \u00e0 membrana celular<\/li>\n<li>O vetor \u00e9 internalizado pela c\u00e9lula em uma ves\u00edcula e transportado<\/li>\n<li>O vetor libera o gene funcional no n\u00facleo<\/li>\n<li>A c\u00e9lula produz a enzima faltante do gene saud\u00e1vel<\/li>\n<li>8. O complemento enzim\u00e1tico completo agora pode decompor os GAGs que, de outra forma, se acumulariam em pessoas com MPS II<\/li>\n<li>A enzima resultante \u00e9 secretada e absorvida por outras c\u00e9lulas<\/li>\n<\/ol>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-816d0ab elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"816d0ab\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>A RGX-121 recebeu as designa\u00e7\u00f5es de Medicamento \u00d3rf\u00e3o, Doen\u00e7a Pedi\u00e1trica Rara e Fast Track da Administra\u00e7\u00e3o de Alimentos e Medicamentos (Food and Drug Administration) dos EUA, assim como a designa\u00e7\u00e3o Medicamento \u00d3rf\u00e3o da Ag\u00eancia Europeia de Medicamentos (EMA).RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, and Fast Track designations from the U.S. Food and Drug Administration, as well as Oprhan Drug Designation from the European Medicines Agency (EMA).<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-a8e9c5c references elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"a8e9c5c\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Refer\u00eancias<\/h3>\n<ol>\n<li>Different approaches. American Society of Gene + Cell Therapy; 2022. <a href=\"https:\/\/patienteducation.asgct.org\/gene-therapy-101\/different-approaches\" target=\"_blank\" rel=\"noopener\">https:\/\/patienteducation.asgct.org\/gene-therapy-101\/different-approaches<\/a>. <\/li>\n<li>Petrich J, Marchese D, Jenkins C, Storey M, Blind J. Gene replacement therapy: a primer for the health-system pharmacist. sJ Pharm Pract. 2020;33(6):846-855. <\/li>\n<li>Adachi H, Hengesbach M, Yu Y-T, Morais P. From antisense RNA to RNA modification: therapeutic potential of RNA-based technologies. Biomedicines. 2021;9(5):550. <\/li>\n<li>Pukenas B, Papisov M, Buss N, et al. Intracisternal administration of AAV9 gene therapies to target the central nervous system. Presented at WORLD<em>Symposium<\/em>\u2122 2021 Virtual Scientific Meeting, February 9, 2021, Poster #207. <\/li>\n<\/ol>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-51c5af4 elementor-section-stretched light-green-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"51c5af4\" data-element_type=\"section\" data-e-type=\"section\" id=\"campsiite-study\" data-settings=\"{&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-89e45fb\" data-id=\"89e45fb\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-4c96548 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"4c96548\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h2>Estudo CAMPSIITE\u2122<\/h2>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-096883b elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"096883b\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Vis\u00e3o geral do estudo cl\u00ednico<\/h3>\n<p>Atualmente, estamos inscrevendo crian\u00e7as com s\u00edndrome de Hunter <strong>neurop\u00e1tica<\/strong> (MPS II) com mais de 4 meses e menos de 5 anos no Estudo CAMPSIITE\u2122, um estudo da RGX-121 multic\u00eantrico, aberto e de grupo \u00fanico de Fase I\/II\/III.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-4d1bbec elementor-widget-divider--view-line elementor-invisible elementor-widget elementor-widget-divider\" data-id=\"4d1bbec\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeIn&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-ea9640d elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"ea9640d\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Participar do CAMPSIITE<\/h3>\n<p>Se seu filho foi diagnosticado com MPS II e sua fam\u00edlia est\u00e1 interessada em participar do Estudo CAMPSIITE, fale com o m\u00e9dico que atende seu filho para MPS II ou entre em contato com um dos centros do estudo. O CAMPSIITE \u00e9 um estudo cl\u00ednico global, que dever\u00e1 incluir centros nos EUA, Brasil e Canad\u00e1. <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-2877502 what-to-expect-map elementor-widget elementor-widget-html\" data-id=\"2877502\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"html.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<div class=\"title-img-wrap\">\n    <h4><img decoding=\"async\" src=\"\/wp-content\/uploads\/2023\/06\/title-pt-br-1.svg\" alt=\"O que esperar\"><\/h4>\n<\/div>\n<div class=\"process-step initial-meeting\">\n    <h5>Reuni\u00e3o inicial com o m\u00e9dico do estudo<\/h5>\n    <p>Os pais de crian\u00e7as que possam ser eleg\u00edveis precisar\u00e3o se reunir com um m\u00e9dico do estudo para revisar os detalhes do estudo e o processo de consentimento livre e esclarecido.<\/p>\n<\/div>\n<div class=\"process-step determine-eligibility\">\n    <h5>Determina\u00e7\u00e3o da elegibilidade<\/h5>\n    <p>O m\u00e9dico do estudo e a equipe falar\u00e3o sobre a sa\u00fade de seu filho, informa\u00e7\u00f5es b\u00e1sicas sobre o estudo e possivelmente solicitar\u00e3o os prontu\u00e1rios m\u00e9dicos.<\/p>\n<\/div>\n<div class=\"process-step if-eligible\">\n    <h5>Se eleg\u00edvel e se voc\u00ea decidir incluir seu filho<\/h5>\n    <p>Se parecer que seu filho pode se qualificar, a equipe do estudo pode programar uma visita de triagem inicial para come\u00e7ar a determinar a elegibilidade e revisar o Termo de Consentimento Livre e Esclarecido com voc\u00ea em detalhes. O processo de determina\u00e7\u00e3o da elegibilidade pode levar algumas semanas. <\/p>\n<\/div>\n<div class=\"process-step study-duration\">\n    <h5>Dura\u00e7\u00e3o do estudo principal<\/h5>\n    <p>Se seu filho se qualificar <em>e<\/em> voc\u00ea decidir inclu\u00ed-lo no estudo, a participa\u00e7\u00e3o na parte principal do estudo durar\u00e1 cerca de 2 anos. Durante esse per\u00edodo, seu filho visitar\u00e1 a cl\u00ednica v\u00e1rias vezes por ano para v\u00e1rios exames e avalia\u00e7\u00f5es. <\/p>\n<\/div>\n<div class=\"process-step follow-up\">\n    <h5>Acompanhamento de longo prazo<\/h5>\n    <p>Ao final do estudo principal, seu filho ser\u00e1 incentivado a ser inclu\u00eddo em um estudo de acompanhamento de longo prazo, que visa compreender a seguran\u00e7a e efic\u00e1cia de longo prazo da RGX-121.<\/p>\n<\/div>\n<div class=\"map-bg\">\n    <svg id=\"eMPFgTbyUiR1\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" viewBox=\"0 0 1121 866\" shape-rendering=\"geometricPrecision\" text-rendering=\"geometricPrecision\"><defs><polygon id=\"eMPFgTbyUiR3\" points=\"0,0 72.501621,0 72.501621,73.365635 0,73.365635\"\/><\/defs><g transform=\"translate(.281189-.855713)\"><g transform=\"translate(0 2.5)\"><g transform=\"translate(0 762.5)\"><path 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class=\"elementor-element elementor-element-0e25d05 ct-callout elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"0e25d05\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Acesse <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" target=\"_blank\" rel=\"noopener\">ClinicalTrials.gov<\/a>, usando o identificador NCT03566043 para obter mais informa\u00e7\u00f5es sobre este estudo, inclusive crit\u00e9rios de elegibilidade.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-f411321 find-a-study elementor-section-boxed elementor-section-height-default elementor-section-height-default elementor-invisible\" 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href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\">Acesse ClinicalTrials.gov (identificador n.\u00ba NCT03566043) para obter uma lista completa dos centros do estudo.<\/a><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-d80affa elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"d80affa\" data-element_type=\"section\" data-e-type=\"section\" id=\"contact-us\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-ee80b9c\" data-id=\"ee80b9c\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element 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data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-f22079c elementor-widget elementor-widget-text-editor\" data-id=\"f22079c\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Se quiser entrar em contato com um membro de nossa equipe de apoio ao paciente, envie um e-mail para <a href=\"mailto:MPSII@regenxbio.com\">MPSII@regenxbio.com<\/a>.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-9498aaa callout-column elementor-invisible\" data-id=\"9498aaa\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-b2c1b4e card elementor-widget elementor-widget-text-editor\" data-id=\"b2c1b4e\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>D\u00favidas espec\u00edficas sobre o estudo<\/h3>\n<p>Para d\u00favidas espec\u00edficas sobre o estudo, como elegibilidade, entre em contato com um centro do estudo. Informa\u00e7\u00f5es de contato dos centros de estudo podem ser encontradas em <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\">ClinicalTrials.gov<\/a> (identificador n.\u00ba NCT03566043). <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-dc72258 card elementor-widget elementor-widget-text-editor\" data-id=\"dc72258\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Somente profissionais da sa\u00fade<\/h3><p>Se for um profissional da sa\u00fade e quiser obter mais informa\u00e7\u00f5es sobre nossos estudos para pacientes com MPS II, entre em contato conosco pelo e-mail <a href=\"mailto:medinfo@regenxbio.com\">medinfo@regenxbio.com<\/a>.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"<p>Um tratamento experimental \u00fanico para mucopolissacaridose tipo II (s\u00edndrome de Hunter) A REGENXBIO, l\u00edder global em terapia g\u00eanica AAV, est\u00e1 atualmente inscrevendo participantes em um estudo cl\u00ednico de RGX-121, uma terapia g\u00eanica experimental \u00fanica para o tratamento potencial da mucopolissacaridose tipo II, tamb\u00e9m conhecida como MPS II ou s\u00edndrome de Hunter. O estudo cl\u00ednico, denominado [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":385,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_genesis_hide_title":false,"_genesis_hide_breadcrumbs":false,"_genesis_hide_singular_image":false,"_genesis_hide_footer_widgets":false,"_genesis_custom_body_class":"","_genesis_custom_post_class":"","_genesis_layout":"","footnotes":""},"class_list":["post-462","page","type-page","status-publish","has-post-thumbnail","entry"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Campsiite Study: MPS II (Hunter Syndrome) Clinical Trial \u2013 REGENXBIO<\/title>\n<meta name=\"description\" content=\"REGENXBIO is enrolling participants in a clinical trial of RGX-121, an investigational, one-time gene therapy for potential treatment of MPS II (Hunter syndrome).\" \/>\n<meta name=\"robots\" content=\"index, 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