{"id":2150,"date":"2025-08-29T20:13:39","date_gmt":"2025-08-29T20:13:39","guid":{"rendered":"https:\/\/www.insiemecontrobatten.it\/?page_id=2150"},"modified":"2026-01-27T21:26:31","modified_gmt":"2026-01-27T21:26:31","slug":"malattia-di-batten","status":"publish","type":"page","link":"https:\/\/www.insiemecontrobatten.it\/en\/malattia-di-batten\/","title":{"rendered":"Batten Disease"},"content":{"rendered":"<div data-elementor-type=\"wp-page\" data-elementor-id=\"2150\" class=\"elementor elementor-2150\">\n\t\t\t\t<div class=\"elementor-element elementor-element-93263f3 e-flex e-con-boxed e-con e-parent\" data-id=\"93263f3\" data-element_type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-6c32ff7 e-flex e-con-boxed e-con e-child\" data-id=\"6c32ff7\" data-element_type=\"container\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t\t\t<div class=\"elementor-element elementor-element-888fcee elementor-widget elementor-widget-heading\" data-id=\"888fcee\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h1 class=\"elementor-heading-title elementor-size-default\">Batten Disease<\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-4cb475b e-flex e-con-boxed e-con e-parent\" data-id=\"4cb475b\" data-element_type=\"container\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-458f621 e-con-full e-flex e-con e-child\" data-id=\"458f621\" data-element_type=\"container\">\n\t\t\t\t<div class=\"elementor-element elementor-element-9dbc1b1 elementor-widget elementor-widget-text-editor\" data-id=\"9dbc1b1\" data-element_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 class=\"translation-block\"><span style=\"font-weight: 400\">Batten disease is the term used to describe the neuronal ceroid lipofuscinoses (NCLs), a group of rare, genetic, and neurodegenerative disorders that affect children and adolescents.<\/span><\/p><p><span style=\"font-weight: 400;\"><br \/><\/span><span style=\"font-weight: 400;\">Sono patologie ereditarie causate da mutazioni in diversi geni (CLN1, CLN2, CLN3 e altri) e si caratterizzano per l\u2019accumulo di sostanze di scarto nei lisosomi delle cellule, con conseguente degenerazione progressiva del sistema nervoso centrale.<\/span><\/p><p class=\"translation-block\"><span style=\"font-weight: 400\">Among the various forms, one of the most common is the variant linked to the CLN3 gene, which represents the \u201cclassic\u201d form of Batten disease.\nThis form typically appears between the ages of 4 and 7 and initially manifests as progressive vision loss, which rapidly advances to blindness. Over time, additional symptoms develop, including epileptic seizures, motor and coordination difficulties, cognitive decline, and loss of language, with a significant impact on both quality of life and life expectancy. <\/span><\/p><p><span style=\"font-weight: 400;\">\u00a0<\/span><\/p><p><span style=\"font-weight: 400;\">La diagnosi si basa principalmente su\u00a0analisi genetiche, integrate da valutazioni neurologiche e oftalmologiche, oltre che da esami strumentali come risonanza magnetica ed elettroencefalogramma.<\/span><\/p><p><span style=\"font-weight: 400;\">\u00a0<\/span><\/p><p><span style=\"font-weight: 400;\">Attualmente\u00a0in Italia ed Europa non esiste una cura risolutiva\u00a0per la malattia di Batten CLN3. I trattamenti disponibili sono di tipo\u00a0sintomatico e di supporto, con l\u2019obiettivo di migliorare la qualit\u00e0 di vita del bambino e della famiglia: farmaci antiepilettici, fisioterapia, logopedia, sostegno psicologico ed educativo.<\/span><\/p><p><span style=\"font-weight: 400;\">\u00a0<\/span><\/p><p class=\"translation-block\"><span style=\"font-weight: 400\">In recent years, however, research has opened new possibilities through the development of innovative therapeutic approaches.\nIn particular, <b>the experimental CLN301 gene therapy <\/b> represents one of the most promising lines of investigation. This strategy uses an adeno-associated viral (AAV) vector to deliver a functional copy of the CLN3 gene into nerve cells, with the goal of compensating for the genetic defect and slowing disease progression.\n\nCLN301 is currently undergoing international clinical trials, and although results are still preliminary, it represents one of the most concrete hopes for the future of individuals affected by this condition.\nThis is the path that <b>HOPE4U INSIEME CONTRO BATTEN APS <\/b> has chosen to pursue, in order to offer young Rachele the future she deserves. <\/span><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-c96eca9 e-flex e-con-boxed e-con e-parent\" data-id=\"c96eca9\" data-element_type=\"container\" data-settings=\"{&quot;background_background&quot;:&quot;classic&quot;}\">\n\t\t\t\t\t<div class=\"e-con-inner\">\n\t\t<div class=\"elementor-element elementor-element-5db1738 e-con-full e-flex e-con e-child\" data-id=\"5db1738\" data-element_type=\"container\">\n\t\t\t\t<div class=\"elementor-element elementor-element-7e98b1d elementor-widget elementor-widget-heading\" data-id=\"7e98b1d\" data-element_type=\"widget\" data-widget_type=\"heading.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<h4 class=\"elementor-heading-title elementor-size-default\">Perch\u00e9 parlarne?<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-728a3ca elementor-widget elementor-widget-text-editor\" data-id=\"728a3ca\" data-element_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 class=\"translation-block\">Raising awareness about this disease means giving a voice to families, supporting research, and promoting increasingly equitable access to care and services.\nThis is the commitment that <b>HOPE4U INSIEME CONTRO BATTEN APS <\/b> carries forward every single day.<\/p>\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-143bd5d elementor-widget elementor-widget-text-editor\" data-id=\"143bd5d\" data-element_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 style=\"font-size: 16px; font-style: normal; font-weight: 400; text-align: center;\">You can support us with a donation via bank transfer:<\/p>\n<p style=\"font-size: 16px; font-style: normal; font-weight: 400; text-align: center;\"><span style=\"font-style: inherit; font-weight: inherit;\">Account holder:&nbsp;<\/span><span style=\"font-weight: bold; font-style: inherit;\">HOPE4U INSIEME CONTRO BATTEN APS<\/span><\/p><p style=\"font-size: 16px; font-style: normal; font-weight: 400; text-align: center;\"><span style=\"font-style: inherit;\">C.F.:&nbsp;<strong>92022830548<\/strong><\/span><span style=\"font-weight: bold; font-style: inherit;\"><\/span><\/p>\n<p style=\"font-size: 16px; font-style: normal; font-weight: 400; text-align: center;\">IBAN:&nbsp;<span style=\"font-weight: bold;\">IT10V0344038480000000266600<\/span><\/p>\n<p style=\"font-size: 16px; font-style: normal; font-weight: 400; text-align: center;\">Bank:&nbsp;<span style=\"font-weight: bold;\">Banco Desio<\/span><\/p>\n<p style=\"font-size: 16px; font-style: normal; font-weight: 400; text-align: center;\">Donation purpose:&nbsp;<span style=\"font-weight: bold;\">Donazione<\/span><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t<div class=\"elementor-element elementor-element-3a154d3 e-con-full e-flex e-con e-child\" data-id=\"3a154d3\" data-element_type=\"container\">\n\t\t\t\t<div class=\"elementor-element elementor-element-9af555f elementor-widget elementor-widget-image\" data-id=\"9af555f\" data-element_type=\"widget\" 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 fetchpriority=\"high\" decoding=\"async\" width=\"246\" height=\"300\" src=\"https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-60x73.png\" class=\"attachment-medium size-medium wp-image-2126\" alt=\"cropped logo raw\" srcset=\"https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-246x300.png 246w, https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-839x1024.png 839w, https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-768x938.png 768w, https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-1258x1536.png 1258w, https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-10x12.png 10w, https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw-60x73.png 60w, https:\/\/www.insiemecontrobatten.it\/wp-content\/uploads\/2025\/08\/cropped-logo_raw.png 1277w\" sizes=\"(max-width: 246px) 100vw, 246px\" title=\"Batten Disease\">\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>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>","protected":false},"excerpt":{"rendered":"<p>La malattia di Batten La\u00a0malattia di Batten\u00a0\u00e8 il termine con cui si indicano le\u00a0ceroidolipofuscinosi neuronali (CNL) un gruppo di malattie genetiche rare e neurodegenerative che colpiscono in et\u00e0 pediatrica e adolescenziale. Sono patologie ereditarie causate da mutazioni in diversi geni (CLN1, CLN2, CLN3 e altri) e si caratterizzano per l\u2019accumulo di sostanze di scarto nei [&hellip;]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"site-sidebar-layout":"no-sidebar","site-content-layout":"page-builder","ast-site-content-layout":"full-width-container","site-content-style":"unboxed","site-sidebar-style":"unboxed","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"disabled","ast-breadcrumbs-content":"","ast-featured-img":"disabled","footer-sml-layout":"","theme-transparent-header-meta":"enabled","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"set","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"class_list":["post-2150","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/pages\/2150","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/comments?post=2150"}],"version-history":[{"count":26,"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/pages\/2150\/revisions"}],"predecessor-version":[{"id":2443,"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/pages\/2150\/revisions\/2443"}],"wp:attachment":[{"href":"https:\/\/www.insiemecontrobatten.it\/en\/wp-json\/wp\/v2\/media?parent=2150"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}