{"id":7974,"date":"2026-05-27T17:33:34","date_gmt":"2026-05-27T14:33:34","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7974"},"modified":"2026-05-27T17:33:35","modified_gmt":"2026-05-27T14:33:35","slug":"dyne-251-z-rostudirsen-phase-3-forzetto-clinical-trial","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/dyne-251-z-rostudirsen-phase-3-forzetto-clinical-trial\/","title":{"rendered":"DYNE-251 Faz 3 FORZETTO Z-Rostudirsen&#039;in DMD i\u00e7in Klinik \u00c7al\u0131\u015fmas\u0131 Resmen Ba\u015flad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Dyne Therapeutics, Duchenne kas distrofisi (DMD) hastas\u0131 aileler i\u00e7in \u00f6nemli bir ad\u0131m daha atarak, Z-Rostudirsen&#039;in DMD i\u00e7in Faz 3 FORZETTO Denemesinin (NCT07608432) ba\u015flad\u0131\u011f\u0131n\u0131 duyurdu.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">K\u00fcresel Faz 3 \u00e7al\u0131\u015fmas\u0131, ekzon 51 atlamas\u0131na uygun olan DMD hastalar\u0131nda z-rostudirsen'i (DYNE-251) de\u011ferlendirecektir. \u015eirket ayr\u0131ca, h\u0131zland\u0131r\u0131lm\u0131\u015f onay i\u00e7in bu \u00e7eyre\u011fin sonlar\u0131nda ABD'ye FDA i\u00e7in Biyolojik \u00dcr\u00fcn Lisans Ba\u015fvurusu (BLA) sunmay\u0131 planlamaktad\u0131r. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/biologics-license-application-bla\/\" target=\"_blank\" data-type=\"post\" data-id=\"5027\" rel=\"noreferrer noopener\">BLA ne anlama geliyor?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FORZETTO klinik \u00e7al\u0131\u015fmas\u0131n\u0131n tasar\u0131m\u0131, \u0130talya&#039;n\u0131n Floransa kentinde d\u00fczenlenecek olan 19. Uluslararas\u0131 N\u00f6rom\u00fcsk\u00fcler Hastal\u0131klar Kongresi&#039;nde (ICNMD) sunulacakt\u0131r.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#what-is-z-rostudirsen-dyne-251\">Z-Rostudirsen (DYNE-251) Nedir?<\/a><\/li><li><a href=\"#how-the-forzetto-phase-3-trial-will-work\">FORZETTO Faz 3 Denemesi Nas\u0131l \u0130lerleyecek?<\/a><\/li><li><a href=\"#primary-endpoint-focuses-on-muscle-function\">Birincil hedef kas fonksiyonuna odaklanmaktad\u0131r<\/a><\/li><li><a href=\"#additional-secondary-endpoints-in-the-forzetto-study\">FORZETTO \u00c7al\u0131\u015fmas\u0131nda Ek \u0130kincil Sonu\u00e7 Noktalar\u0131<\/a><\/li><li><a href=\"#fda-alignment-and-accelerated-approval-plans\">FDA Hedefleri ve H\u0131zland\u0131r\u0131lm\u0131\u015f Onay Planlar\u0131<\/a><\/li><li><a href=\"#why-the-dyne-therapeutics-phase-3-forzetto-trial-of-z-rostudirsen-for-dmd-matters\">DMD i\u00e7in Z-Rostudirsen&#039;in Faz 3 FORZETTO \u00c7al\u0131\u015fmas\u0131n\u0131n \u00d6nemi<\/a><\/li><li><a href=\"#contact-information\">\u0130leti\u015fim Bilgileri<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"what-is-z-rostudirsen-dyne-251\" class=\"wp-block-heading\">Z-Rostudirsen (DYNE-251) Nedir?<\/h2>\n\n\n\n<h3 id=\"a-next-generation-exon-51-skipping-therapy\" class=\"wp-block-heading\">Yeni Nesil Ekzon 51 Atlama Terapisi<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Z-rostudirsen, di\u011fer ad\u0131yla DYNE-251, ekzon 51 atlamas\u0131ndan fayda g\u00f6rebilecek DMD mutasyonlar\u0131na sahip ki\u015filer i\u00e7in tasarlanm\u0131\u015f, ara\u015ft\u0131rma a\u015famas\u0131nda olan bir tedavidir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu tedavi, v\u00fccudun kas dokular\u0131nda ve merkezi sinir sisteminde neredeyse tam uzunlukta distrofin proteini \u00fcretmesine yard\u0131mc\u0131 olmay\u0131 ama\u00e7lamaktad\u0131r. Distrofin, kaslar\u0131 hasardan korumak i\u00e7in gereklidir ve yoklu\u011fu DMD hastalar\u0131nda ilerleyici kas g\u00fc\u00e7s\u00fczl\u00fc\u011f\u00fcne yol a\u00e7ar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Dyne Therapeutics'e g\u00f6re, tedavinin amac\u0131 sadece laboratuvar biyobelirte\u00e7lerini iyile\u015ftirmek de\u011fil, ayn\u0131 zamanda hastalar\u0131n daha iyi hareket etmelerine, daha iyi nefes almalar\u0131na ve g\u00fcnl\u00fck ya\u015fam kalitelerini art\u0131rmalar\u0131na yard\u0131mc\u0131 olmakt\u0131r.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"847\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg\" alt=\"Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 51 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar\" class=\"wp-image-4526\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg 847w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-248x300.jpg 248w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-768x929.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1270x1536.jpg 1270w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1693x2048.jpg 1693w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-10x12.jpg 10w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-347x420.jpg 347w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-150x181.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-300x363.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-696x842.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1068x1292.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1920x2323.jpg 1920w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping.jpg 1984w\" sizes=\"auto, (max-width: 847px) 100vw, 847px\" \/><figcaption class=\"wp-element-caption\"><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><\/figcaption><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u015e\u0130MD\u0130 DENEY\u0130N:<\/strong> <strong><a href=\"https:\/\/dmdwarrior.com\/tr\/duchenne-exon-deletion-tool\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD mi yoksa BMD mi? Ekzon Kontrol Arac\u0131<\/a><\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"how-the-forzetto-phase-3-trial-will-work\" class=\"wp-block-heading\">FORZETTO Faz 3 Denemesi Nas\u0131l \u0130lerleyecek?<\/h2>\n\n\n\n<h3 id=\"global-randomized-placebo-controlled-study\" class=\"wp-block-heading\">K\u00fcresel Rastgelele\u015ftirilmi\u015f Plasebo Kontroll\u00fc \u00c7al\u0131\u015fma<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>DYNE-251 Faz 3 FORZETTO \u00c7al\u0131\u015fmas\u0131, Z-rostudirsen'in g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015f, k\u00fcresel, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc bir \u00e7al\u0131\u015fmad\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fman\u0131n temel ayr\u0131nt\u0131lar\u0131 \u015funlard\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Yakla\u015f\u0131k 90 ayakta tedavi g\u00f6ren erkek kat\u0131l\u0131mc\u0131<\/li>\n\n\n\n<li>4 ila 18 ya\u015f aras\u0131<\/li>\n\n\n\n<li>Kat\u0131l\u0131mc\u0131lar\u0131n 51. ekzonun atlanmas\u0131na raz\u0131 olmalar\u0131 gerekmektedir.<\/li>\n\n\n\n<li>Hastalar a\u015fa\u011f\u0131daki se\u00e7eneklerden birini alacaklard\u0131r:\n<ul class=\"wp-block-list\">\n<li>20 mg\/kg z-rostudirsen<\/li>\n\n\n\n<li>veya plasebo<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Tedavi d\u00f6rt haftada bir (Q4W) uygulanacakt\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0130lk klinik ara\u015ft\u0131rma merkezi zaten faaliyete ge\u00e7ti ve kay\u0131tlar ba\u015flad\u0131.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"primary-endpoint-focuses-on-muscle-function\" class=\"wp-block-heading\">Birincil hedef kas fonksiyonuna odaklanmaktad\u0131r<\/h2>\n\n\n\n<h3 id=\"rise-from-floor-velocity-will-be-measured\" class=\"wp-block-heading\">Zeminden Y\u00fckselme H\u0131z\u0131 \u00d6l\u00e7\u00fclecektir<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n temel amac\u0131, 73. haftada yerden y\u00fckselme h\u0131z\u0131ndaki (RFF) veya y\u00fckselme s\u00fcresi (TTR) h\u0131z\u0131ndaki de\u011fi\u015fiklikleri \u00f6l\u00e7mektir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu test, hastan\u0131n yerden ne kadar h\u0131zl\u0131 kalkabildi\u011fini \u00f6l\u00e7er ve DMD klinik \u00e7al\u0131\u015fmalar\u0131nda kas g\u00fcc\u00fc ve motor koordinasyonunun \u00f6nemli bir g\u00f6stergesi olarak kabul edilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha \u00f6nceki Faz 1\/2 DELIVER \u00e7al\u0131\u015fmas\u0131nda, z-rostudirsen ile tedavi edilen hastalarda, alt\u0131 ay sonra RFF h\u0131z\u0131nda plasebo ile tedavi edilen kat\u0131l\u0131mc\u0131lara k\u0131yasla anlaml\u0131 iyile\u015fmeler g\u00f6zlemlenmi\u015ftir.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"additional-secondary-endpoints-in-the-forzetto-study\" class=\"wp-block-heading\">FORZETTO \u00c7al\u0131\u015fmas\u0131nda Ek \u0130kincil Sonu\u00e7 Noktalar\u0131<\/h2>\n\n\n\n<h3 id=\"researchers-will-track-mobility-and-lung-function\" class=\"wp-block-heading\">Ara\u015ft\u0131rmac\u0131lar Hareketlili\u011fi ve Akci\u011fer Fonksiyonunu Takip Edecekler<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma ayr\u0131ca a\u015fa\u011f\u0131dakiler de dahil olmak \u00fczere \u00e7e\u015fitli ek fonksiyonel sonu\u00e7lar\u0131 de\u011ferlendirecektir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Ad\u0131m H\u0131z\u0131 95. Y\u00fczdelik Dilim (SV95C)<\/li>\n\n\n\n<li><a href=\"https:\/\/dmdwarrior.com\/tr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"4959\" rel=\"noreferrer noopener\">Kuzey Y\u0131ld\u0131z\u0131 Ayaktan De\u011ferlendirme (NSAA)<\/a><\/li>\n\n\n\n<li>10 Metre Y\u00fcr\u00fcy\u00fc\u015f\/Ko\u015fu (10MWR)<\/li>\n\n\n\n<li>D\u00f6rt Basamakl\u0131 T\u0131rman\u0131\u015f (4SC)<\/li>\n\n\n\n<li>Zorlu Vital Kapasite Y\u00fczdesi Tahmini (FVC%p)<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar ayr\u0131ca, tedavinin g\u00fcnl\u00fck ya\u015fam\u0131 nas\u0131l etkileyebilece\u011fini daha iyi anlamak i\u00e7in hastalar\u0131n bildirdi\u011fi sonu\u00e7lar\u0131 da toplayacaklar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0130lk 72 haftal\u0131k plasebo kontroll\u00fc d\u00f6nemin ard\u0131ndan, uygun kat\u0131l\u0131mc\u0131lar 96 haftal\u0131k a\u00e7\u0131k etiketli bir uzatma \u00e7al\u0131\u015fmas\u0131na devam edebilirler.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"fda-alignment-and-accelerated-approval-plans\" class=\"wp-block-heading\">FDA Hedefleri ve H\u0131zland\u0131r\u0131lm\u0131\u015f Onay Planlar\u0131<\/h2>\n\n\n\n<h3 id=\"forzetto-could-support-full-traditional-approval\" class=\"wp-block-heading\">FORZETTO, Geleneksel Onay S\u00fcrecinin Tamam\u0131n\u0131 Destekleyebilir<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Dyne Therapeutics, \u015firketin FORZETTO Faz 3 deneme tasar\u0131m\u0131 ve protokol\u00fc konusunda ABD G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA) ile uyum sa\u011flad\u0131\u011f\u0131n\u0131 belirtti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fma, Amerika Birle\u015fik Devletleri&#039;nde h\u0131zland\u0131r\u0131lm\u0131\u015f onay s\u00fcrecini tam ve geleneksel onay s\u00fcrecine d\u00f6n\u00fc\u015ft\u00fcrmek i\u00e7in gerekli olan do\u011frulay\u0131c\u0131 deneme niteli\u011findedir. \u00c7al\u0131\u015fma ayr\u0131ca ABD d\u0131\u015f\u0131ndaki gelecekteki d\u00fczenleyici ba\u015fvurular\u0131 da destekleyebilir.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-the-dyne-therapeutics-phase-3-forzetto-trial-of-z-rostudirsen-for-dmd-matters\" class=\"wp-block-heading\">DMD i\u00e7in Z-Rostudirsen&#039;in Faz 3 FORZETTO \u00c7al\u0131\u015fmas\u0131n\u0131n \u00d6nemi<\/h2>\n\n\n\n<h3 id=\"a-potentially-important-development-for-the-dmd-community\" class=\"wp-block-heading\">DMD Toplulu\u011fu \u0130\u00e7in Potansiyel Olarak \u00d6nemli Bir Geli\u015fme<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Duchenne kas distrofisi (DMD) i\u00e7in Z-Rostudirsen'in Dyne Therapeutics Faz 3 FORZETTO \u00c7al\u0131\u015fmas\u0131n\u0131n ba\u015flat\u0131lmas\u0131, \u00f6zellikle ekzon 51 atlama tedavilerine uygun hastalar i\u00e7in Duchenne toplulu\u011fu a\u00e7\u0131s\u0131ndan \u00f6nemli bir d\u00f6n\u00fcm noktas\u0131d\u0131r.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Umut vadeden \u00f6nceki klinik veriler ve \u015fu anda devam eden b\u00fcy\u00fck bir do\u011frulama \u00e7al\u0131\u015fmas\u0131yla birlikte, ara\u015ft\u0131rmac\u0131lar ve aileler, DYNE-251&#039;nin Duchenne kas distrofisi olan ki\u015filer i\u00e7in anlaml\u0131 fonksiyonel iyile\u015fmeler sa\u011flay\u0131p sa\u011flayamayaca\u011f\u0131n\u0131 yak\u0131ndan izleyecekler.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"contact-information\" class=\"wp-block-heading\">\u0130leti\u015fim Bilgileri<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Ad\u0131: Dyne Klinik Ara\u015ft\u0131rmalar\u0131<\/li>\n\n\n\n<li>Telefon Numaras\u0131: +1-781-317-1919<\/li>\n\n\n\n<li>E-posta: clinicaltrials@dyne-tx.com<\/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 has announced the start of the Dyne Therapeutics Phase 3 FORZETTO Trial (NCT07608432) of Z-Rostudirsen for DMD, marking another major step forward for families affected by Duchenne muscular dystrophy (DMD). The global Phase 3 study will evaluate z-rostudirsen (DYNE-251) in people living with DMD who are amenable to exon 51 skipping. The company [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8013,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,143,208,145,122,193,421,194,780,460],"class_list":["post-7974","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","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-forzetto","tag-z-rostudirsen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7974","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=7974"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7974\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8013"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7974"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7974"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7974"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}