{"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":"erste-zugelassene-therapie-fur-rett-syndrom-eine-potenzielle-hoffnung-fur-patienten-mit-mecp2-duplikationssyndrom","status":"publish","type":"post","link":"https:\/\/dupmecp2.eu\/de\/erste-zugelassene-therapie-fur-rett-syndrom-eine-potenzielle-hoffnung-fur-patienten-mit-mecp2-duplikationssyndrom\/","title":{"rendered":"Erste zugelassene Behandlung f\u00fcr das Rett-Syndrom: eine potenzielle Hoffnung f\u00fcr Patienten mit MECP2-Duplikationssyndrom?"},"content":{"rendered":"<p>Der 10. M\u00e4rz 2023 stellt ein sehr wichtiges Datum in der Gemeinschaft der seltenen Krankheiten dar.<\/p>\n\n\n\n<p>Trofinetide wurde von der FDA zugelassen zur Behandlung von Patienten mit <a href=\"#rett\" target=\"_blank\" rel=\"noreferrer noopener\">Rett-Syndrom<\/a>Damit ist es die erste Behandlung f\u00fcr das Rett-Syndrom \u00fcberhaupt. Das Medikament, das unter dem Namen Daybue\u2122 vermarktet wird, wird voraussichtlich Ende April 2023 in den USA f\u00fcr Patienten, die \u00e4lter als zwei Jahre sind, kommerziell erh\u00e4ltlich sein.<\/p>\n\n\n\n<p>Trofinetide ist ein Tripeptid, das von einem Protein namens Insulin-like growth factor 1 (IGF-1) abgeleitet ist. Es kann oral verabreicht werden. Das Medikament soll Entz\u00fcndungen hemmen und die Kommunikation zwischen den Neuronen im Gehirn verbessern, die bei Menschen mit Rett-Syndrom beeintr\u00e4chtigt ist. Phase-III-Klinikversuche haben gezeigt, dass Trofinetide verschiedene Symptome des Rett-Syndroms verbessert, wie z. B. die Verbesserung der sozialen Kommunikation, des Verhaltens und der Handfunktion.<\/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\">Read more about 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>W\u00e4hrend die zugrundeliegende genetische Ursache des MECP2-Duplikationssyndroms (MDS) sich von derjenigen des <a href=\"#rett\">Rett-Syndrom<\/a>Bei beiden Erkrankungen liegt eine Mutation des MECP2-Gens vor. Rett-Syndrom betrifft vor allem M\u00e4dchen und hat zu wenig aktives MeCP2-Protein, w\u00e4hrend MDS, das vor allem bei Jungen diagnostiziert wird, zu h\u00f6heren Werten des Proteins f\u00fchrt. Interessanterweise verursachen sowohl Rett als auch MDS eine beeintr\u00e4chtigte Gehirnentwicklung und -funktion, was zu \u00e4hnlichen Symptomen wie geistiger Behinderung, Koordinationsproblemen und Krampfanf\u00e4llen f\u00fchrt.<\/p>\n\n\n\n<p>Daher tragen wissenschaftliche Fortschritte beim Rett-Syndrom dazu bei, das Wissen \u00fcber die anderen Syndrome zu vergr\u00f6\u00dfern. The approval of Daybue\u2122 opens the door to further research into the use of Trofinetide in patients with MECP2 duplication syndrome.<\/p>\n\n\n\n<p>Der Kampf um das MECP2-Duplikationssyndrom ist noch nicht vorbei. Helfen Sie mit, die Forschung zur Behandlung von MDS voranzutreiben, indem Sie spenden, das Bewusstsein f\u00fcr das Syndrom sch\u00e4rfen und sich an unseren Aktionen beteiligen!<\/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\/de\/become-a-member\/?lang=en\" style=\"border-radius:12px;background-color:#f7a13f\">Schlie\u00dfen Sie sich unserem Kampf an!<\/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\/de\/erste-zugelassene-therapie-fur-rett-syndrom-eine-potenzielle-hoffnung-fur-patienten-mit-mecp2-duplikationssyndrom\/?lang=en\" \/>\n<meta property=\"og:locale\" content=\"de_DE\" \/>\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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