{"id":7875,"date":"2026-05-08T10:37:41","date_gmt":"2026-05-08T07:37:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7875"},"modified":"2026-05-08T10:37:43","modified_gmt":"2026-05-08T07:37:43","slug":"bmn-351-exon-51-skipping-therapy-phase-2-clinical-trial-nct07573631","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/bmn-351-exon-51-skipping-therapy-phase-2-clinical-trial-nct07573631\/","title":{"rendered":"BioMarin, Duchenne Musk\u00fcler Distrofisi i\u00e7in BMN 351 Ekzon 51 Atlama Tedavisi Faz 2 Klinik \u00c7al\u0131\u015fmas\u0131n\u0131 Ba\u015flatt\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>BioMarin Pharmaceutical, Duchenne kas distrofisi (DMD) hastalar\u0131 i\u00e7in tasarlanm\u0131\u015f, deneysel bir ekzon 51 atlama tedavisi olan BMN 351'i de\u011ferlendiren yeni bir Faz 2 uzatma \u00e7al\u0131\u015fmas\u0131n\u0131 (NCT07573631) resmen ba\u015flatt\u0131.<\/strong> Bu \u00e7al\u0131\u015fma, daha \u00f6nce 351-201 klinik \u00e7al\u0131\u015fmas\u0131n\u0131 tamamlam\u0131\u015f kat\u0131l\u0131mc\u0131larda uzun vadeli g\u00fcvenlik, tolerans ve fiziksel i\u015flev sonu\u00e7lar\u0131na odaklanacakt\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">BMN 351 Ekzon 51 Atlama Tedavisi Faz 2 Klinik \u00c7al\u0131\u015fmas\u0131 Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>BMN 351 ekzon 51 atlama terapisi faz 2 klinik \u00e7al\u0131\u015fmas\u0131, ekzon 51 atlamaya uygun Duchenne kas distrofisi olan bireyler i\u00e7in \u00e7ok merkezli, a\u00e7\u0131k etiketli bir uzatma \u00e7al\u0131\u015fmas\u0131d\u0131r.<\/strong> Ara\u015ft\u0131rmac\u0131lar, haftal\u0131k intraven\u00f6z BMN 351 inf\u00fczyonlar\u0131n\u0131n daha uzun bir tedavi s\u00fcresi boyunca ne kadar g\u00fcvenli ve etkili kald\u0131\u011f\u0131n\u0131 de\u011ferlendirecekler.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Deneme \u00e7al\u0131\u015fmas\u0131na, daha \u00f6nce 351-201 olarak bilinen Faz 1\/2 \u00e7al\u0131\u015fmas\u0131n\u0131 tamamlam\u0131\u015f olan 4 ila 23 ya\u015f aras\u0131 en fazla 18 kat\u0131l\u0131mc\u0131n\u0131n dahil edilmesi planlan\u0131yor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Duchenne Kas Distrofisi ve Ekzon 51 Atlama Hakk\u0131nda Bilgiler<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Duchenne Kas Distrofisi Nedir?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne kas distrofisi, ilerleyici kas g\u00fc\u00e7s\u00fczl\u00fc\u011f\u00fcne ve kas dejenerasyonuna neden olan nadir bir genetik hastal\u0131kt\u0131r. Sa\u011fl\u0131kl\u0131 kas h\u00fccrelerinin korunmas\u0131 i\u00e7in gerekli olan distrofin genindeki mutasyonlardan kaynaklan\u0131r.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Ekzon 51 Atlama Tedavisi Nedir?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Ekzon atlama terapileri, v\u00fccudun distrofin proteininin daha k\u0131sa ancak i\u015flevsel bir versiyonunu \u00fcretmesine yard\u0131mc\u0131 olmak i\u00e7in tasarlanm\u0131\u015ft\u0131r. Ekzon 51 atlama, \u00f6zellikle protein \u00fcretim s\u00fcrecinde ekzon 51&#039;in atlanmas\u0131ndan potansiyel olarak fayda g\u00f6rebilecek mutasyonlara sahip hastalar\u0131 hedef al\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">BMN 351, uygun hastalarda hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatmaya yard\u0131mc\u0131 olabilecek yeni nesil bir ekzon atlama tedavisi olarak ara\u015ft\u0131r\u0131lmaktad\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\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\">Faz 2 \u00c7al\u0131\u015fmas\u0131na \u0130li\u015fkin \u00d6nemli Ayr\u0131nt\u0131lar<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">\u00c7al\u0131\u015fma Tasar\u0131m\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik deneme \u015fu \u015fekildedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c7ok merkezli<\/li>\n\n\n\n<li>A\u00e7\u0131k etiketli<\/li>\n\n\n\n<li>Uzun s\u00fcreli uzatma \u00e7al\u0131\u015fmas\u0131<\/li>\n\n\n\n<li>Haftal\u0131k intraven\u00f6z dozlamaya odaklan\u0131lm\u0131\u015ft\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fman\u0131n ilk ziyareti, daha \u00f6nceki 351-201 denemesinin son ziyareti olarak da i\u015flev g\u00f6recektir.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Tahmini Zaman \u00c7izelgesi<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c7al\u0131\u015fman\u0131n Ba\u015flang\u0131\u00e7 Tarihi: Nisan 2026<\/li>\n\n\n\n<li>Birincil Tamamlama: Aral\u0131k 2031<\/li>\n\n\n\n<li>Tahmini \u00c7al\u0131\u015fma Tamamlanma Tarihi: Aral\u0131k 2031<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">\u00c7al\u0131\u015fmaya Kat\u0131lan \u00dclkeler<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma, a\u015fa\u011f\u0131daki klinik ara\u015ft\u0131rma merkezlerini kapsamaktad\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0130talya<\/li>\n\n\n\n<li>Hollanda<\/li>\n\n\n\n<li>\u0130spanya<\/li>\n\n\n\n<li>T\u00fcrkiye<\/li>\n\n\n\n<li>Birle\u015fik Krall\u0131k<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Kimler Kat\u0131labilir?<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">Ba\u015fl\u0131ca Dahil Edilme Kriterleri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Kat\u0131l\u0131mc\u0131lar\u0131n \u015funlar\u0131 yapmas\u0131 gerekmektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>351-201 \u00e7al\u0131\u015fmas\u0131n\u0131 tamamlamak<\/li>\n\n\n\n<li>Stabil kortikosteroid tedavisine devam etmek<\/li>\n\n\n\n<li>\u00c7al\u0131\u015fma ziyareti gerekliliklerine uymak<\/li>\n\n\n\n<li>Gerekti\u011finde bilgilendirilmi\u015f onam veya r\u0131za vermek<\/li>\n\n\n\n<li>Protokol y\u00f6nergeleri gerektiriyorsa do\u011fum kontrol y\u00f6ntemleri kullanmak<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">Ba\u015fl\u0131ca Hari\u00e7 Tutma Kriterleri<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">A\u015fa\u011f\u0131daki durumlarda kat\u0131l\u0131mc\u0131lar kat\u0131lamaz:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>P\u0131ht\u0131la\u015fma bozukluklar\u0131 biliniyorsa<\/li>\n\n\n\n<li>Yasakl\u0131 ila\u00e7lar kullan\u0131l\u0131yorsa<\/li>\n\n\n\n<li>Ba\u015flang\u0131\u00e7 de\u011ferlendirmesinden \u00f6nceki 12 hafta i\u00e7inde onaylanm\u0131\u015f bir ba\u015fka ekzon atlama tedavisi daha ald\u0131ysan\u0131z<\/li>\n\n\n\n<li>Daha \u00f6nce DMD i\u00e7in gen terapisi ald\u0131ysan\u0131z<\/li>\n\n\n\n<li>Antikoag\u00fclanlar veya imm\u00fcnosupresanlar kulland\u0131ysan\u0131z<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Bu Klinik Ara\u015ft\u0131rma Duchenne Toplulu\u011fu \u0130\u00e7in Neden \u00d6nemli?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>BMN 351 ekzon 51 atlama terapisi faz 2 klinik denemesinin ba\u015flat\u0131lmas\u0131, Duchenne kas distrofisi i\u00e7in hedefe y\u00f6nelik genetik terapilerin geli\u015ftirilmesinde bir di\u011fer \u00f6nemli ad\u0131m\u0131 temsil etmektedir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Uzun s\u00fcreli yayg\u0131nla\u015ft\u0131rma \u00e7al\u0131\u015fmalar\u0131 \u00f6zellikle \u00f6nemlidir \u00e7\u00fcnk\u00fc ara\u015ft\u0131rmac\u0131lara \u015fu konularda yard\u0131mc\u0131 olurlar:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Tedavinin faydalar\u0131n\u0131n zaman i\u00e7inde korunup korunamayaca\u011f\u0131<\/li>\n\n\n\n<li>Uzun vadeli g\u00fcvenlik profilleri<\/li>\n\n\n\n<li>G\u00fcnl\u00fck fiziksel i\u015flevler \u00fczerindeki etkiler<\/li>\n\n\n\n<li>Hastal\u0131\u011f\u0131n ilerlemesi \u00fczerindeki potansiyel etki<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Duchenne kas distrofisinden etkilenen aileler i\u00e7in, ekzon atlama ara\u015ft\u0131rmalar\u0131ndaki s\u00fcrekli ilerleme, gelecekteki tedavi se\u00e7enekleri konusunda yeni bir umut \u0131\u015f\u0131\u011f\u0131 sa\u011fl\u0131yor.<\/p>\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>BioMarin Pharmaceutical has officially launched a new Phase 2 extension study (NCT07573631) evaluating BMN 351, an investigational exon 51 skipping therapy designed for people living with Duchenne muscular dystrophy (DMD). The study will focus on long-term safety, tolerability, and physical function outcomes in participants who previously completed the earlier 351-201 clinical trial. What Is the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7877,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[759,760,137,122,193,421,194],"class_list":["post-7875","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-biomarin","tag-bmn-351","tag-clinical-trial","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7875","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=7875"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7875\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/7877"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7875"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7875"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7875"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}