{"id":15701,"date":"2023-03-12T19:22:19","date_gmt":"2023-03-12T19:22:19","guid":{"rendered":"https:\/\/dupmecp2.eu\/?p=15701"},"modified":"2023-03-15T20:51:15","modified_gmt":"2023-03-15T20:51:15","slug":"il-primo-trattamento-approvato-per-la-sindrome-di-rett-rappresenta-una-potenziale-speranza-per-i-pazienti-con-sindrome-da-duplicazione-di-mecp2","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/it\/il-primo-trattamento-approvato-per-la-sindrome-di-rett-rappresenta-una-potenziale-speranza-per-i-pazienti-con-sindrome-da-duplicazione-di-mecp2\/","title":{"rendered":"Primo trattamento approvato per la sindrome di Rett: una potenziale speranza per i pazienti con sindrome da duplicazione di MECP2?"},"content":{"rendered":"<p>Il 10 marzo 2023 segna una data molto importante per la comunit\u00e0 delle malattie rare.<\/p>\n\n\n\n<p>La trofinetide \u00e8 stata approvata dalla FDA per il trattamento di pazienti con <a href=\"#rett\" target=\"_blank\" rel=\"noreferrer noopener\">Sindrome di Rett<\/a>che lo rende il primo trattamento in assoluto per la sindrome di Rett. Il farmaco, commercializzato con il nome di Daybue\u2122, dovrebbe essere disponibile in commercio negli Stati Uniti entro la fine di aprile 2023 per i pazienti di et\u00e0 superiore ai 2 anni.<\/p>\n\n\n\n<p>La trofinetide \u00e8 un tripeptide derivato da una proteina chiamata fattore di crescita insulino-simile 1 (IGF-1). Pu\u00f2 essere somministrato per via orale. Si ritiene che questo farmaco riduca l'infiammazione e migliori la comunicazione tra i neuroni del cervello, che \u00e8 compromessa nei soggetti con sindrome di Rett. Gli studi clinici di fase III hanno dimostrato che la trofinetide migliora vari sintomi della sindrome di Rett, come la comunicazione sociale, il comportamento e la funzione delle mani.<\/p>\n\n\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"\" style=\"border-radius:12px;background-color:#f7a13f\" target=\"_blank\" rel=\"noreferrer noopener\">Per saperne di pi\u00f9 sull'Acadia<\/a><\/div>\n<\/div>\n\n\n\n<div style=\"height:20px\" aria-hidden=\"true\" class=\"wp-block-spacer\"><\/div>\n\n\n\n<p>Sebbene la causa genetica alla base della sindrome da duplicazione di MECP2 (MDS) sia diversa da quella di <a href=\"#rett\">Sindrome di Rett<\/a>Entrambi i disturbi comportano una mutazione del gene MECP2. La sindrome di Rett colpisce soprattutto le bambine e presenta una quantit\u00e0 insufficiente di proteina MeCP2 attiva, mentre la MDS, diagnosticata soprattutto nei maschi, comporta livelli pi\u00f9 elevati della proteina. \u00c8 interessante notare che sia la Rett che la MDS causano un'alterazione dello sviluppo e della funzione cerebrale, con sintomi simili, come disabilit\u00e0 intellettiva, problemi di coordinazione e convulsioni.<\/p>\n\n\n\n<p>Pertanto, i progressi scientifici sulla sindrome di Rett contribuiscono ad aumentare le conoscenze sulle altre. L'approvazione di Daybue\u2122 apre le porte a ulteriori ricerche sull'uso della trofinetide nei pazienti con sindrome da duplicazione MECP2.<\/p>\n\n\n\n<p>La lotta per la sindrome da duplicazione MECP2 non \u00e8 finita. Contribuite a far progredire la ricerca su una cura per la SMD con donazioni, sensibilizzazione sulla sindrome e unendovi alle nostre azioni!<\/p>\n\n\n\n<div class=\"wp-block-buttons is-layout-flex wp-block-buttons-is-layout-flex\">\n<div class=\"wp-block-button\"><a class=\"wp-block-button__link has-background wp-element-button\" href=\"https:\/\/dupmecp2.eu\/it\/become-a-member\/?lang=en\" style=\"border-radius:12px;background-color:#f7a13f\">Unisciti alla nostra lotta!<\/a><\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>The 10th of March 2023 sets a very important date in the rare disease community. Trofinetide has been approved by the FDA for treatment of patients with Rett Syndrome, making [&hellip;]<\/p>","protected":false},"author":1,"featured_media":15703,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[87],"tags":[],"class_list":["post-15701","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v25.8 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>First approved treatment for Rett syndrome: a potential hope for patients with MECP2 duplication syndrome? - DupMECP2<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/dupmecp2.eu\/it\/il-primo-trattamento-approvato-per-la-sindrome-di-rett-rappresenta-una-potenziale-speranza-per-i-pazienti-con-sindrome-da-duplicazione-di-mecp2\/?lang=en\" \/>\n<meta property=\"og:locale\" content=\"it_IT\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"First approved treatment for Rett syndrome: a potential hope for patients with MECP2 duplication syndrome? - DupMECP2\" \/>\n<meta property=\"og:description\" content=\"The 10th of March 2023 sets a very important date in the rare disease community. 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