{"id":3218,"date":"2025-03-17T10:34:47","date_gmt":"2025-03-17T07:34:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3218"},"modified":"2025-03-17T10:39:51","modified_gmt":"2025-03-17T07:39:51","slug":"dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics, Duchenne Musk\u00fcler Distrofi&#039;de DYNE-251&#039;nin 1\/2. A\u015fama DELIVER Denemesinden 18 Ay Boyunca Benzeri Olmayan ve S\u00fcrd\u00fcr\u00fclebilir Fonksiyonel \u0130yile\u015fme Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Genetik kaynakl\u0131 n\u00f6rom\u00fcsk\u00fcler hastal\u0131klar\u0131 olan ki\u015filer i\u00e7in ya\u015fam\u0131 de\u011fi\u015ftiren tedavi y\u00f6ntemlerini geli\u015ftirmeye odaklanan klinik a\u015famadaki bir \u015firket olan Dyne Therapeutics, bug\u00fcn DYNE-251&#039;nin devam eden Faz 1\/2 DELIVER denemesinden elde edilen yeni uzun vadeli klinik verilerin, se\u00e7ilen 20 mg\/kg Q4W (yakla\u015f\u0131k PMO dozu) kay\u0131t dozunda benzeri g\u00f6r\u00fclmemi\u015f ve s\u00fcrd\u00fcr\u00fclebilir i\u015flevsel iyile\u015fme g\u00f6sterdi\u011fini duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DELIVER \u00e7al\u0131\u015fmas\u0131, ekzon 51 atlamaya uygun Duchenne kas distrofisi (DMD) hastalar\u0131nda DYNE-251&#039;yi de\u011ferlendiriyor ve \u00e7al\u0131\u015fmadan elde edilen g\u00fcncel sonu\u00e7lar bu hafta 2025 Kas Distrofisi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda sunulacak.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DELIVER Faz 1\/2 deneme sunumunun temel bulgular\u0131 \u015funlard\u0131r:<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0130\u015flev:<\/strong> 20 mg\/kg (se\u00e7ilen kay\u0131t dozu) ve 10 mg\/kg1 DYNE-251 Q4W kohortlar\u0131nda s\u0131ras\u0131yla 12 ve 18 ay boyunca, ba\u015flang\u0131\u00e7 seviyesinden itibaren birden fazla i\u015flevsel son noktada anlaml\u0131 ve s\u00fcrd\u00fcr\u00fclebilir iyile\u015fmeler g\u00f6zlemlendi. \u0130\u015flevsel de\u011ferlendirmeler aras\u0131nda Ad\u0131m H\u0131z\u0131 95. Centil (SV95C), North Star Ambulatuvar De\u011ferlendirmesi (NSAA), 10 Metrelik Y\u00fcr\u00fcy\u00fc\u015f\/Ko\u015fu S\u00fcresi (10-MWR) ve Yerden Kalkma S\u00fcresi yer ald\u0131.\n<ul class=\"wp-block-list\">\n<li>6 ayl\u0131k zaman noktas\u0131ndan itibaren, DELIVER&#039;\u0131n hem 10 mg\/kg hem de 20 mg\/kg kohortlar\u0131nda g\u00f6zlemlenen ba\u015flang\u0131\u00e7 seviyesinden SV95C de\u011fi\u015fimi, yay\u0131nlanm\u0131\u015f \u00f6nerilen minimal klinik olarak \u00f6nemli fark\u0131 (MCID) a\u015ft\u0131.<\/li>\n\n\n\n<li>SV95C, hastalar\u0131n normal g\u00fcnl\u00fck ortamlar\u0131ndaki ambulatuvar performans\u0131n\u0131n dijital objektif bir sonu\u00e7 \u00f6l\u00e7\u00fcs\u00fcd\u00fcr ve Avrupa&#039;daki DMD klinik deneyleri i\u00e7in birincil sonlan\u0131m noktas\u0131 olarak kabul edilmektedir.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Distrofin ekspresyonu:<\/strong> Daha \u00f6nce bildirildi\u011fi gibi, DYNE-251, ekzon 51 atlamaya uygun DMD&#039;li hastalarda Western blot ile \u00f6l\u00e7\u00fcld\u00fc\u011f\u00fcnde benzeri g\u00f6r\u00fclmemi\u015f neredeyse tam uzunlukta distrofin ekspresyonu g\u00f6sterdi. 6 ayl\u0131k zaman noktas\u0131nda, 20 mg\/kg DYNE-251 Q4W ile tedavi edilen hastalar\u0131n ortalama mutlak distrofin ekspresyonu normalin 8.72%&#039;siydi (kas i\u00e7eri\u011fine g\u00f6re ayarlanm\u0131\u015f). Dyne, ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nin distrofini h\u0131zland\u0131r\u0131lm\u0131\u015f onay i\u00e7in bir vekil biyobelirte\u00e7 olarak kullanma emsalinin hala mevcut oldu\u011funu do\u011frulad\u0131.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>G\u00fcvenlik ve tolere edilebilirlik:<\/strong> DYNE-251, DELIVER \u00e7al\u0131\u015fmas\u0131na kat\u0131lan 54 kat\u0131l\u0131mc\u0131ya g\u00f6re olumlu bir g\u00fcvenlik profili g\u00f6stermi\u015ftir. 21 Kas\u0131m 2024 itibar\u0131yla sa\u011flanan \u00f6nceki g\u00fcncellemeden bu yana, g\u00fcvenlik profili de\u011fi\u015fmeden kalm\u0131\u015ft\u0131r ve tedaviyle ili\u015fkili yeni ciddi olumsuz olaylar g\u00f6zlemlenmemi\u015ftir.\n<ul class=\"wp-block-list\">\n<li>\u00c7al\u0131\u015fma ilac\u0131n\u0131n 970 dozu, 77,1 hasta y\u0131l\u0131 takip s\u00fcresi boyunca bug\u00fcne kadar uyguland\u0131 ve baz\u0131 hastalar ~2,5 y\u0131la kadar takip edildi.<\/li>\n\n\n\n<li>\u00c7al\u0131\u015fma ilac\u0131n\u0131n 20 mg\/kg doz d\u00fczeyindeki 546 dozu bug\u00fcne kadar uyguland\u0131.<\/li>\n<\/ul>\n<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics, a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data from its ongoing Phase 1\/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg\/kg Q4W (approximate PMO dose). The DELIVER trial is [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3221,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194],"class_list":["post-3218","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3218","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=3218"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3218\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3221"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3218"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3218"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3218"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}