{"id":425,"date":"2022-08-10T14:19:11","date_gmt":"2022-08-10T18:19:11","guid":{"rendered":"https:\/\/campsiitestudy.com\/home\/"},"modified":"2023-06-09T09:58:38","modified_gmt":"2023-06-09T13:58:38","slug":"home","status":"publish","type":"page","link":"https:\/\/campsiitestudy.com\/fr\/","title":{"rendered":"Home"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"425\" class=\"elementor elementor-425 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-318\" 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-291\" 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-294\" 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-300\" 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>Traitement exp\u00e9rimental \u00e0 dose unique de la mucopolysaccharidose de type II (syndrome de Hunter)<\/h1>\n<p>REGENXBIO, un chef de file mondial de la th\u00e9rapie g\u00e9nique AAV, recrute actuellement des participants \u00e0 un essai clinique visant \u00e0 \u00e9valuer RGX-121, une th\u00e9rapie g\u00e9nique exp\u00e9rimentale \u00e0 dose unique pour le traitement potentiel de la mucopolysaccharidose de type II, \u00e9galement connue sous le nom de MPS II ou syndrome de Hunter. L\u2019essai clinique, appel\u00e9 \u00ab \u00c9tude CAMPSIITE\u2122 \u00bb, \u00e9valuera l\u2019effet de RGX-121 chez des gar\u00e7ons atteints de MPS II<sup>1<\/sup> neuronopathique \u00e2g\u00e9s de 4 mois \u00e0 5 ans. <\/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\">En savoir plus sur <span class=\"large-cta-text\">CAMPSIITE<\/span><\/a><\/p><p><a class=\"text-link\" href=\"#contact-us\"><span class=\"large-cta-text\">Nous Contacter<\/span><\/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<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-303\" 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-297\" 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-306\" 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-309\" 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-288\" 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>\u00c0 propos du syndrome 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>Qu\u2019est-ce que la mucopolysaccharidose de type 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>La MPS II, ou syndrome de Hunter, est une maladie g\u00e9n\u00e9tique rare qui survient dans environ une naissance masculine sur 100 000 \u00e0 170 000.<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-fr-1.svg\" class=\"attachment-full size-full wp-image-550\" 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>Les personnes atteintes de MPS II pr\u00e9sentent une mutation du g\u00e8ne responsable de la fabrication d\u2019une enzyme appel\u00e9e iduronate-2-sulfatase (<em>IDS<\/em>). <sup>1<\/sup><\/li>\n<li>Les cellules ont besoin de cette enzyme pour d\u00e9composer de longues cha\u00eenes de mol\u00e9cules de sucre appel\u00e9es glycosaminoglycanes (GAG). <sup>1<\/sup><\/li>\n<li>Si les GAG s\u2019accumulent dans les cellules du corps, ils peuvent causer des dommages progressifs \u00e0 de nombreux organes, y compris le cerveau, les oreilles, les poumons, le c\u0153ur, l\u2019estomac, les intestins, les os, les articulations, la bouche et la peau. <sup>2<\/sup><\/li>\n<li>Les dommages r\u00e9sultant de l\u2019accumulation de GAG dans les cellules sont irr\u00e9versibles, ce qui souligne l\u2019importance d\u2019un diagnostic et d\u2019un traitement pr\u00e9coces. <sup>4<\/sup><\/li>\n<li>Les traitements approuv\u00e9s actuels pour le syndrome de Hunter ne traitent pas compte des effets de la MPS II sur le cerveau. <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>Cellule saine vs Cellule avec 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>CELLULE SAINE<\/h3>\n<ol>\n<li>Glycosaminoglycanes (GAG) Mol\u00e9cules de sucre r\u00e9sultant de fonctions cellulaires normales<\/li>\n<li>L\u2019ADN normal code pour diverses enzymes qui d\u00e9composent les GAG<\/li>\n<\/ol>\n<p>Ces enzymes sont dans le lysosome, et non en suspension dans le cytoplasme &#8211; d&rsquo;o\u00f9 le trouble de stockage lysosomal<\/p>\n<ol start=\"3\">\n<li>Les enzymes d\u00e9composent les GAG<\/li>\n<\/ol>\n<h3>CELLULE AVEC MPS II NEURONOPATHIQUE<\/h3>\n<ol start=\"4\">\n<li>L\u2019ADN contient un code erron\u00e9 pour la production d\u2019une enzyme sp\u00e9cifique qui d\u00e9compose les GAG<\/li>\n<\/ol>\n<p>Cytoplasme (dans le lysosome plut\u00f4t que dans le cytoplasme)<\/p>\n<ol start=\"5\">\n<li>Les enzymes ne d\u00e9composent que partiellement les GAG, ce qui entra\u00eene leur accumulation dans la cellule<\/li>\n<li> Chez les patients atteints de MPS II neuronopathique, l\u2019accumulation de GAG entra\u00eene des l\u00e9sions tissulaires et organiques dans tout l\u2019organisme, y compris une neurod\u00e9g\u00e9n\u00e9rescence<\/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-Canadian-French.jpg\" data-elementor-open-lightbox=\"yes\" data-elementor-lightbox-title=\"A Canadian French\" data-e-action-hash=\"#elementor-action%3Aaction%3Dlightbox%26settings%3DeyJpZCI6NDk5LCJ1cmwiOiJodHRwczpcL1wvY2FtcHNpaXRlc3R1ZHkuY29tXC93cC1jb250ZW50XC91cGxvYWRzXC8yMDIzXC8wNlwvQS1DYW5hZGlhbi1GcmVuY2guanBnIn0%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-Canadian-French.jpg\" class=\"attachment-full size-full wp-image-499\" alt=\"\" srcset=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Canadian-French.jpg 2000w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Canadian-French-300x144.jpg 300w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Canadian-French-1024x491.jpg 1024w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Canadian-French-768x368.jpg 768w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/A-Canadian-French-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>Types de MPS II<\/h3>\n<p>En g\u00e9n\u00e9ral, il existe deux types de MPS II, qui varient consid\u00e9rablement en termes de gravit\u00e9, de taux de progression et d\u2019\u00e2ge d\u2019apparition. <\/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>Att\u00e9nu\u00e9e<\/h4>\n<p>Dans la MPS II att\u00e9nu\u00e9e, la maladie progresse lentement, avec des effets minimes ou nuls sur le cerveau ou le d\u00e9veloppement intellectuel<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>Neuronopathique<\/h4>\n<p>Dans la MPS II neuronopathique, \u00e9galement connue sous le nom de MPS II grave, la progression de la maladie est plus rapide et a des effets significatifs sur le cerveau, ainsi que sur le d\u00e9veloppement intellectuel.<sup>1<\/sup> La MPS II neuronopathique repr\u00e9sente jusqu\u2019\u00e0 70 % <sup>2<\/sup> des cas de la maladie <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>Dans la MPS II grave, ou la forme neuronopathique, l\u2019enfant peut atteindre les premiers stades de d\u00e9veloppement, mais le rythme d\u2019acquisition des comp\u00e9tences ralentit t\u00f4t dans le d\u00e9veloppement (g\u00e9n\u00e9ralement entre 12 et 24 mois apr\u00e8s la naissance). Ce retard devient plus apparent avec le temps; les capacit\u00e9s neurod\u00e9veloppementales (apprentissage et compr\u00e9hension, langage expressif) et la motricit\u00e9 fine se situant en dehors de la plage normale entre 24 et 29 mois. L\u2019acquisition des comp\u00e9tences atteint g\u00e9n\u00e9ralement un plateau vers l\u2019\u00e2ge de 5 \u00e0 6 ans, l\u2019enfant fonctionnant alors \u00e0 un niveau de d\u00e9veloppement similaire \u00e0 celui d\u2019un enfant de 2 \u00e0 3 ans non atteint de la maladie<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>Les activit\u00e9s neurod\u00e9veloppementales comprennent :<\/p><ul><li>Apprentissage<\/li><li>Compr\u00e9hension<\/li><li>Langage expressif<\/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>Sympt\u00f4mes<\/h3><p>Les enfants atteints de MPS II peuvent pr\u00e9senter des sympt\u00f4mes divers, en fonction de l\u2019\u00e2ge d\u2019apparition et de la gravit\u00e9 de la maladie: <sup>1,2,6<\/sup><\/p><ul><li>Infections r\u00e9currentes de l\u2019oreille<\/li><li>Hernie ombilicale ou inguinale (renflement d\u2019une partie de l\u2019intestin \u00e0 travers l\u2019ouverture des muscles abdominaux ou dans la r\u00e9gion de l\u2019aine)<\/li><li>Hydroc\u00e9phalie (accumulation de liquide dans le cerveau)<\/li><li>\u00c9paississement des narines et des l\u00e8vres<\/li><li>Macroc\u00e9phalie (t\u00eate plus grosse que la normale)<\/li><li>Macroglossie (langue plus grosse que la normale)<\/li><li>H\u00e9patospl\u00e9nom\u00e9galie (hypertrophie de la rate et du foie)<\/li><li>Perte auditive<\/li><li>Hypertrophie des amygdales<\/li><li>Voix rauque<\/li><li>Troubles du sommeil<\/li><li>Petite taille<\/li><li>Raideur dans les articulations et limitation des mouvements articulaires<\/li><li>Syndrome du canal carpien<\/li><li>Retard de croissance<\/li><li>Apparition tardive des dents ou espaces importants entre les dents<\/li><li>Excroissances blanches sur la peau<\/li><li>D\u00e9clin du d\u00e9veloppement neurologique, d\u00e9ficience intellectuelle<\/li><li>Probl\u00e8mes de comportement (hyperactivit\u00e9, probl\u00e8mes d\u2019attention, destructivit\u00e9, absence de crainte)<\/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>Ressources additionnelles<\/h3><p>Pour obtenir plus d\u2019informations au sujet de la MPS II, consultez les liens suivants:<\/p><ul><li><a href=\"https:\/\/mpssociety.org\/\" target=\"_blank\" rel=\"noopener\"> National MPS Society (U.S.) (anglais)<\/a><\/li><li><a href=\"https:\/\/projectalive.org\/\" target=\"_blank\" rel=\"noopener\"> Project Alive (anglais)<\/a><\/li><li><a href=\"https:\/\/rarediseases.org\/rare-diseases\/mucopolysaccharidosis-type-ii-2\/\" target=\"_blank\" rel=\"noopener\"> National Organization for Rare Disorders (NORD) (anglais)<\/a><\/li><li><a href=\"https:\/\/casahunter.org.br\/home-en\/\" target=\"_blank\" rel=\"noopener\">Casa Hunter (Br\u00e9sil) (anglais, portugais)<\/a><\/li><li><a href=\"https:\/\/www.mpssociety.ca\/\" target=\"_blank\" rel=\"noopener\"> Canadian MPS Society for Mucopolysaccharide &amp; Related Diseases (anglais)<\/a><\/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 (tch\u00e8que, n\u00e9erlandais, anglais, fran\u00e7ais, allemand, italien, polonais, portugais, espagnol)<\/a><\/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>Ressources additionnelles<\/h3><p>Pour obtenir plus d\u2019informations au sujet de la MPS II, consultez les liens suivants:<\/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 (U.S.) (anglais)<\/a><\/p>\n<p><a href=\"https:\/\/projectalive.org\/\" target=\"_blank\" rel=\"noopener\"> Project Alive (anglais)<\/a><\/p>\n<p><a href=\"https:\/\/rarediseases.org\/rare-diseases\/mucopolysaccharidosis-type-ii-2\/\" target=\"_blank\" rel=\"noopener\"> National Organization for Rare Disorders (NORD) (anglais)<\/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-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 (Br\u00e9sil) (anglais, portugais)<\/a><\/p>\n<p><a href=\"https:\/\/www.mpssociety.ca\/\" target=\"_blank\" rel=\"noopener\"> Canadian MPS Society for Mucopolysaccharide &amp; Related Diseases (anglais)<\/a><\/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 (tch\u00e8que, n\u00e9erlandais, anglais, fran\u00e7ais, allemand, italien, polonais, portugais, espagnol)<\/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<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>R\u00e9f\u00e9rences:<\/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. Pr\u00e9sent\u00e9 lors du symposium annuel 2022 de la Society for the Study of Inborn Errors of Metabolism (SSIEM), Fribourg, Allemagne, ao\u00fbt 2022. R\u00e9sum\u00e9 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>\u00c0 propos de la th\u00e9rapie g\u00e9nique 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>Que sont les g\u00e8nes et la th\u00e9rapie g\u00e9nique?<sup>1-3<\/sup><\/h3>\n<p>Chaque cellule du corps humain contient des chromosomes constitu\u00e9s de g\u00e8nes. Ces g\u00e8nes sont constitu\u00e9s d\u2019ADN, qui fournit des instructions indiquant \u00e0 l\u2019organisme comment fabriquer des prot\u00e9ines, qui constituent des \u00e9l\u00e9ments essentiels de notre corps. Le corps humain contient des milliers de g\u00e8nes.  <\/p>\n<p>La th\u00e9rapie g\u00e9nique est un traitement qui implique l\u2019introduction, l\u2019\u00e9limination ou la modification de mat\u00e9riel g\u00e9n\u00e9tique dans les cellules d\u2019une personne. Il existe plusieurs approches en mati\u00e8re de th\u00e9rapie g\u00e9nique : <\/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>Addition de g\u00e8ne<\/h4>\n<p>L\u2019ajout d\u2019un g\u00e8ne \u00e0 une personne, permettant au corps de cette personne de produire une prot\u00e9ine fonctionnelle (aussi parfois appel\u00e9 remplacement de g\u00e8ne). RGX-121 utilise cette approche. <\/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>\u00c9dition g\u00e9n\u00e9tique<\/h4><p>L\u2019insertion, le remplacement ou la suppression d\u2019ADN d\u00e9fectueux dans un g\u00e8ne.<sup>2-3<\/sup><\/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<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>Modification de l\u2019ARN<\/h4>\n<p>La modification de l\u2019ARN (une mol\u00e9cule semblable \u00e0 l\u2019ADN qui aide la cellule \u00e0 cr\u00e9er des prot\u00e9ines \u00e0 partir des g\u00e8nes) dans le but de bloquer la production d\u2019une prot\u00e9ine dysfonctionnelle provenant d\u2019un g\u00e8ne mut\u00e9.<\/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>Qu\u2019est-ce que la technologie NAV<sup>\u00ae<\/sup>?<\/h3>\n<p>Les th\u00e9rapies g\u00e9niques de REGENXBIO font l\u2019objet d\u2019essais cliniques et sont con\u00e7ues pour d\u00e9livrer des g\u00e8nes fonctionnels aux cellules \u00e0 l\u2019aide de vecteurs viraux ad\u00e9no-associ\u00e9s (AAV) comme \u00ab v\u00e9hicules \u00bb d\u2019administration. Les vecteurs AAV sont des virus modifi\u00e9s qui ne peuvent pas augmenter leur nombre ni se reproduire, et qui ne sont pas connus pour provoquer des maladies chez l\u2019homme. <\/p>\n<p>Nos th\u00e9rapies g\u00e9niques exp\u00e9rimentales utilisent toutes des vecteurs viraux issus de notre plateforme exclusive d\u2019administration de g\u00e8nes, que nous appelons la plateforme technologique NAV<sup>\u00ae<\/sup>. Des milliers de patients ont re\u00e7u des th\u00e9rapies g\u00e9niques AAV d\u00e9riv\u00e9es de notre plateforme technologique 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=\"Lecteur vid\u00e9o 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>Qu\u2019est-ce que RGX-121?<\/h3>\n<p>RGX-121 est une th\u00e9rapie g\u00e9nique exp\u00e9rimentale \u00e0 dose unique destin\u00e9e au traitement potentiel de la MPS II. Elle est con\u00e7ue pour utiliser le vecteur NAV<sup>\u00ae<\/sup> AAV9, dans le but de d\u00e9livrer le g\u00e8ne humain de l\u2019iduronate-2-sulfatase (<em>IDS<\/em>) directement dans le liquide entourant le cerveau, appel\u00e9 liquide c\u00e9phalo-rachidien (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>Lorsque le vecteur NAV<sup>\u00ae<\/sup> AAV9 transmet le g\u00e8ne IDS aux cellules du cerveau, <strong>celles-ci peuvent commencer \u00e0 produire l\u2019enzyme I2S n\u00e9cessaire<\/strong> et pourraient constituer une source permanente d\u2019I2S dans le syst\u00e8me nerveux central (SNC), qui comprend le cerveau et la moelle \u00e9pini\u00e8re.<\/li>\n<li>Une production continue de l\u2019enzyme <strong>pourrait pr\u00e9venir le d\u00e9clin du d\u00e9veloppement neurologique<\/strong> chez les enfants qui re\u00e7oivent la th\u00e9rapie g\u00e9nique en d\u00e9but de vie, et pourrait emp\u00eacher l\u2019aggravation des dommages chez les enfants qui re\u00e7oivent la th\u00e9rapie g\u00e9nique \u00e0 un \u00e2ge plus avanc\u00e9.<\/li>\n<li>Contrairement aux traitements actuellement disponibles pour la MPS II, RGX-121 peut traverser la barri\u00e8re h\u00e9mato-enc\u00e9phalique (BHE), un r\u00e9seau protecteur de vaisseaux sanguins et de cellules qui s\u00e9pare le SNC du reste du corps. En traversant la BHE \u00e0 partir du SNC pour atteindre le reste de l\u2019organisme, RGX-121 <strong>pourrait s\u2019attaquer aux effets syst\u00e9miques<\/strong> (ceux qui touchent l\u2019organisme dans son ensemble) de la MPS II4. <sup><\/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>Comment RGX-121 est-il cens\u00e9 agir?<\/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-Canadian-French.jpg\" data-elementor-open-lightbox=\"yes\" data-elementor-lightbox-title=\"B Canadian French\" data-e-action-hash=\"#elementor-action%3Aaction%3Dlightbox%26settings%3DeyJpZCI6NTAzLCJ1cmwiOiJodHRwczpcL1wvY2FtcHNpaXRlc3R1ZHkuY29tXC93cC1jb250ZW50XC91cGxvYWRzXC8yMDIzXC8wNlwvQi1DYW5hZGlhbi1GcmVuY2guanBnIn0%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-Canadian-French.jpg\" class=\"attachment-full size-full wp-image-503\" alt=\"\" srcset=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Canadian-French.jpg 2000w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Canadian-French-300x164.jpg 300w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Canadian-French-1024x561.jpg 1024w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Canadian-French-768x421.jpg 768w, https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/06\/B-Canadian-French-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>Transporteur (vecteur viral ad\u00e9no-associ\u00e9, AAV)<\/h3>\n<ol>\n<li>L\u2019ADN viral est \u00e9limin\u00e9<\/li>\n<li>Un g\u00e8ne <em>IDS<\/em> humain fonctionnel est ins\u00e9r\u00e9<\/li>\n<li>Le traitement est administr\u00e9 dans le syst\u00e8me nerveux central, guid\u00e9 par TDM pendant que le patient est endormi.<\/li>\n<li>Le vecteur se lie \u00e0 la membrane cellulaire<\/li>\n<li>Le vecteur est internalis\u00e9 par la cellule dans une v\u00e9sicule (un petit sac liquide) et transport\u00e9 jusqu\u2019au noyau<\/li>\n<li>Le vecteur lib\u00e8re le g\u00e8ne fonctionnel dans le noyau<\/li>\n<li>La cellule produit l\u2019enzyme manquante \u00e0 partir d\u2019un g\u00e8ne sain<\/li>\n<li>Le compl\u00e9ment enzymatique complet peut maintenant d\u00e9composer les GAG qui s\u2019accumuleraient autrement chez les personnes atteintes de MPS II<\/li>\n<li>L\u2019enzyme qui en r\u00e9sulte est s\u00e9cr\u00e9t\u00e9e et absorb\u00e9e par d\u2019autres cellules.<\/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>RGX-121 a re\u00e7u les d\u00e9signations de m\u00e9dicament orphelin, de maladie p\u00e9diatrique rare et le statut de \u00ab d\u00e9signation acc\u00e9l\u00e9r\u00e9e \u00bb de la Food and Drug Administration des \u00c9tats-Unis, ainsi que la d\u00e9signation de m\u00e9dicament orphelin de l\u2019Agence europ\u00e9enne des m\u00e9dicaments (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>R\u00e9f\u00e9rences<\/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. Pr\u00e9sent\u00e9 lors de la r\u00e9union scientifique virtuelle WORLD<em>Symposium<\/em>\u2122 2021, le 9 f\u00e9vrier 2021, affiche #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>\u00c9tude 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>Aper\u00e7u de l\u2019essai<\/h3>\n<p>Nous recrutons actuellement des enfants atteints du syndrome <strong>neuronopathique<\/strong> de Hunter (MPS II) \u00e2g\u00e9s de plus de 4 mois et de moins de 5 ans, dans le cadre de l\u2019\u00e9tude CAMPSIITE\u2122, une \u00e9tude de phase I\/II\/III, multicentrique, ouverte et \u00e0 groupe unique visant \u00e0 \u00e9valuer RGX-121.<\/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>Participer \u00e0 CAMPSIITE<\/h3>\n<p>Si votre enfant a re\u00e7u un diagnostic de MPS II et que votre famille est int\u00e9ress\u00e9e \u00e0 participer \u00e0 l\u2019\u00e9tude CAMPSIITE, veuillez en discuter avec le m\u00e9decin qui traite votre enfant pour la MPS II, ou communiquez avec l\u2019un des emplacements de l\u2019\u00e9tude. CAMPSIITE est un essai mondial qui devrait inclure des centres aux \u00c9tats-Unis, au Br\u00e9sil et au Canada. <\/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-fr-1.svg\" alt=\"\u00c0 quoi s\u2019attendre\"><\/h4>\n<\/div>\n<div class=\"process-step initial-meeting\">\n    <h5>Premi\u00e8re rencontre avec le m\u00e9decin de l\u2019\u00e9tude<\/h5>\n    <p>Les parents des enfants susceptibles d\u2019\u00eatre admissibles devront consulter un m\u00e9decin de l\u2019\u00e9tude pour passer en revue les d\u00e9tails de l\u2019\u00e9tude et le processus de consentement \u00e9clair\u00e9.<\/p>\n<\/div>\n<div class=\"process-step determine-eligibility\">\n    <h5>D\u00e9termination de l\u2019admissibilit\u00e9<\/h5>\n    <p>Le m\u00e9decin de l\u2019\u00e9tude et l\u2019\u00e9quipe de l\u2019\u00e9tude discuteront de la sant\u00e9 de votre enfant, des renseignements de base concernant l\u2019\u00e9tude et demanderont \u00e9ventuellement des dossiers m\u00e9dicaux.<\/p>\n<\/div>\n<div class=\"process-step if-eligible\">\n    <h5>En cas d\u2019admissibilit\u00e9 et que vous d\u00e9cidez d\u2019inscrire votre enfant<\/h5>\n    <p>S\u2019il s\u2019av\u00e8re que votre enfant est admissible, l\u2019\u00e9quipe de l\u2019\u00e9tude peut organiser une visite de s\u00e9lection initiale pour commencer \u00e0 d\u00e9terminer l\u2019admissibilit\u00e9 et examiner en d\u00e9tail le formulaire de consentement \u00e9clair\u00e9 avec vous. Le processus de d\u00e9termination de l\u2019admissibilit\u00e9 peut prendre quelques semaines. <\/p>\n<\/div>\n<div class=\"process-step study-duration\">\n    <h5>Dur\u00e9e de l'\u00e9tude principale<\/h5>\n    <p>Si votre enfant remplit les conditions requises <em>et<\/em> vous d\u00e9cidez de l\u2019inscrire \u00e0 l\u2019\u00e9tude, la participation \u00e0 la partie principale de l\u2019\u00e9tude durera environ 2 ans. Au cours de cette p\u00e9riode, votre enfant se rendra \u00e0 la clinique plusieurs fois par an pour des tests et \u00e9valuations divers. <\/p>\n<\/div>\n<div class=\"process-step follow-up\">\n    <h5>Suivi \u00e0 long terme<\/h5>\n    <p>\u00c0 la fin de l\u2019\u00e9tude principale, votre enfant sera encourag\u00e9 \u00e0 s\u2019inscrire \u00e0 une \u00e9tude de suivi \u00e0 long terme, qui vise \u00e0 comprendre l\u2019innocuit\u00e9 et l\u2019efficacit\u00e9 \u00e0 long terme de 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>Consultez le site <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" target=\"_blank\" rel=\"noopener\">ClinicalTrials.gov<\/a> en utilisant l\u2019identifiant NCT03566043 pour obtenir plus de renseignements au sujet de cette \u00e9tude, y compris les crit\u00e8res d\u2019admissibilit\u00e9.<\/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\" data-id=\"f411321\" data-element_type=\"section\" data-e-type=\"section\" id=\"campsiite-locations\" 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-top-column elementor-element elementor-element-306c346 card\" data-id=\"306c346\" 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-b447801 elementor-widget elementor-widget-text-editor\" data-id=\"b447801\" 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>Trouvez un centre d\u2019\u00e9tude pr\u00e8s de chez vous!<\/h3>\n<p>L\u2019\u00e9tude CAMPSIITE\u2122 devrait inclure des centres aux \u00c9tats-Unis, au Br\u00e9sil et au Canada.<\/p>\n<p><a class=\"button\" href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\">Consultez le site ClinicalTrials.gov (identifiant no NCT03566043) pour obtenir une liste compl\u00e8te des centres<\/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 elementor-element-e3b09e7 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"e3b09e7\" 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>Nous Contacter<\/h2>\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-7848df7 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"7848df7\" 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-6a79c26 narrow-col elementor-invisible\" data-id=\"6a79c26\" 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-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>Si vous souhaitez communiquer avec un membre de notre \u00e9quipe de d\u00e9fense des patients, vous pouvez nous envoyer un courriel \u00e0: <a href=\"mailto:MPSII@regenxbio.com\">MPSII@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<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>Questions propres \u00e0 l\u2019\u00e9tude<\/h3>\n<p>Pour les questions propres \u00e0 l\u2019\u00e9tude, telles que l\u2019admissibilit\u00e9, veuillez communiquer avec un centre d\u2019\u00e9tude. Les coordonn\u00e9es des centres d\u2019\u00e9tude sont disponibles sur <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\">ClinicalTrials.gov<\/a> (identifiant no 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>Prestataires de soins de sant\u00e9<\/h3><p>Si vous \u00eates un professionnel de la sant\u00e9 et que vous souhaitez obtenir plus de renseignements sur nos \u00e9tudes pour les patients atteints de MPS II, veuillez communiquer avec nous via l\u2019adresse courriel suivante: <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>Traitement exp\u00e9rimental \u00e0 dose unique de la mucopolysaccharidose de type II (syndrome de Hunter) REGENXBIO, un chef de file mondial de la th\u00e9rapie g\u00e9nique AAV, recrute actuellement des participants \u00e0 un essai clinique visant \u00e0 \u00e9valuer RGX-121, une th\u00e9rapie g\u00e9nique exp\u00e9rimentale \u00e0 dose unique pour le traitement potentiel de la mucopolysaccharidose de type II, \u00e9galement [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":384,"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":{"0":"post-425","1":"page","2":"type-page","3":"status-publish","4":"has-post-thumbnail","6":"entry"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - 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, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/campsiitestudy.com\/fr\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Campsiite Study: MPS II (Hunter Syndrome) Clinical Trial \u2013 REGENXBIO\" \/>\n<meta property=\"og: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 property=\"og:url\" content=\"https:\/\/campsiitestudy.com\/fr\/\" \/>\n<meta property=\"og:site_name\" content=\"CAMPSIITE Study\" \/>\n<meta property=\"article:modified_time\" content=\"2023-06-09T13:58:38+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2023\/01\/campsiite-share-1024x538.jpg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" 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