{"id":5295,"date":"2025-12-08T16:05:39","date_gmt":"2025-12-08T13:05:39","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5295"},"modified":"2025-12-08T16:08:05","modified_gmt":"2025-12-08T13:08:05","slug":"dyne-therapeutics-to-apply-for-accelerated-approval-in-q2-2026-for-exon-51-skipping-therapy-dyne-251-z-rostudirsen","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-to-apply-for-accelerated-approval-in-q2-2026-for-exon-51-skipping-therapy-dyne-251-z-rostudirsen\/","title":{"rendered":"Dyne Therapeutics, Exon 51 Atlama Terapisi (DYNE-251) i\u00e7in 2026'n\u0131n 2. \u00c7eyre\u011finde H\u0131zland\u0131r\u0131lm\u0131\u015f Onay i\u00e7in Ba\u015fvuruda Bulunacak"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics, ekzon 51 atlamaya uygun Duchenne kas distrofisi (DMD) hastalar\u0131nda zeleciment rostudirsen&#039;i (z-rostudirsen, ayn\u0131 zamanda DYNE-251 olarak da bilinir) de\u011ferlendiren Faz 1\/2 DELIVER denemesinin Kay\u0131t Geni\u015fletme Kohortu&#039;ndan (REC) elde edilen olumlu ilk sat\u0131r sonu\u00e7lar\u0131n\u0131 duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u015eimdi Ke\u015ffedin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ekzon 51 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251 Klinik Ara\u015ft\u0131rmas\u0131n\u0131n Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kay\u0131t Geni\u015fleme Kohortu (REC) birincil son noktaya ula\u015ft\u0131 ve 6 ayda distrofin seviyesinde 5.46%&#039;ye kadar istatistiksel olarak anlaml\u0131 bir art\u0131\u015f g\u00f6sterdi (kas i\u00e7eri\u011fi ayarl\u0131; p&lt;0,0001), daha \u00f6nce kay\u0131t dozunda g\u00f6zlemlenen ba\u015flang\u0131\u00e7 seviyesinden ayn\u0131 7 kat de\u011fi\u015fimi tekrarlad\u0131 \u2013<\/li>\n\n\n\n<li><strong>REC&#039;de 6. ayda birden fazla klinik son noktada fonksiyonel iyile\u015fme g\u00f6zlemlendi; akci\u011fer fonksiyonu 6. ayda korundu \u2013<\/strong><\/li>\n\n\n\n<li>DELIVER denemesinden elde edilen yeni olumlu uzun vadeli sonu\u00e7lar, 24 ay boyunca de\u011ferlendirilen t\u00fcm son noktalarda s\u00fcrd\u00fcr\u00fclebilir i\u015flevsel iyile\u015fme oldu\u011funu g\u00f6sterdi.<\/li>\n\n\n\n<li><strong>Olumlu g\u00fcvenlik ve tolere edilebilirlik profilinin devam etmesi \u2013<\/strong><\/li>\n\n\n\n<li>ABD H\u0131zland\u0131r\u0131lm\u0131\u015f Onay\u0131 i\u00e7in ba\u015fvurular 2026&#039;n\u0131n 2. \u00e7eyre\u011finde yolunda gidiyor \u2013<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1\/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Discover Now: Mutations and Deletions Amenable to Exon 51 Skipping Therapies Results of DYNE-251 Clinical Trial Follow [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5297,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,122,193,421,194,460],"class_list":["post-5295","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-z-rostudirsen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5295","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5295"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5295\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5297"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5295"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5295"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5295"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}