{"id":4762,"date":"2025-10-10T15:17:22","date_gmt":"2025-10-10T12:17:22","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4762"},"modified":"2025-11-03T20:18:13","modified_gmt":"2025-11-03T17:18:13","slug":"wve-n531-exon-skipping-therapy-phase-2-clinical-trial-nct07209332","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/wve-n531-exon-skipping-therapy-phase-2-clinical-trial-nct07209332\/","title":{"rendered":"Duchenne Musk\u00fcler Distrofisi i\u00e7in WVE-N531 Ekzon Atlama Terapisi Faz 2 Klinik \u00c7al\u0131\u015fmas\u0131 Ba\u015fl\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Wave Life Sciences Ltd., ClinicalTrials.gov web sitesinde WVE-N531'in (Ekson 53 Atlama Terapisi) faz 2 klinik \u00e7al\u0131\u015fmas\u0131n\u0131n \u00dcrd\u00fcn ve Birle\u015fik Krall\u0131k'ta 175 kat\u0131l\u0131mc\u0131yla Kas\u0131m 2025'te ba\u015flayaca\u011f\u0131n\u0131 duyurdu.<\/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-wve-n-531-exon-skipping-therapy\">WVE-N531 Ekzon Atlama Terapisi Nedir?<\/a><\/li><li><a href=\"#wve-n-531-exon-skipping-therapy-phase-2-nct-07209332\">WVE-N531 Ekzon Atlama Terapisi Faz 2 \u2013 NCT07209332<\/a><\/li><li><a href=\"#how-to-participate-in-the-wve-n-531-clinical-trial\">WVE-N531 Klinik Denemesine Nas\u0131l Kat\u0131l\u0131rs\u0131n\u0131z?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-wve-n-531-exon-skipping-therapy\">WVE-N531 Ekzon Atlama Terapisi Nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>WVE-N531, ekzon 53 atlamaya uygun DMD hastalar\u0131 i\u00e7in potansiyel bir tedavi olarak geli\u015ftirilmekte olan ara\u015ft\u0131rma a\u015famas\u0131ndaki bir ekzon atlama tedavisidir.<\/strong> WVE-N531, PN omurga kimyas\u0131 da dahil olmak \u00fczere Wave&#039;in s\u0131n\u0131f\u0131n\u0131n en iyisi oligon\u00fckleotid kimyas\u0131 modifikasyonlar\u0131 kullan\u0131larak tasarlanm\u0131\u015ft\u0131r. WVE-N531, ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nden Yetim \u0130la\u00e7 Tan\u0131m\u0131 ve Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 alm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-53-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 53 Atlamaya Uygun Delesyonlar ve Mutasyonlar<\/a><\/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\/11\/amenable-to-exon-53-skipping-TR.jpg\" alt=\"Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 53 Atlamaya Uygun Delesyonlar ve Mutasyonlar\" class=\"wp-image-4566\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR.jpg 1984w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-248x300.jpg 248w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-847x1024.jpg 847w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-768x929.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-1270x1536.jpg 1270w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-1693x2048.jpg 1693w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-10x12.jpg 10w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-347x420.jpg 347w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-150x181.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-300x363.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-696x842.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-1068x1292.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/amenable-to-exon-53-skipping-TR-1920x2323.jpg 1920w\" sizes=\"auto, (max-width: 847px) 100vw, 847px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"wve-n-531-exon-skipping-therapy-phase-2-nct-07209332\">WVE-N531 Ekzon Atlama Terapisi Faz 2 \u2013 NCT07209332<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu, DMD&#039;li ve WVE-N531 ile ilgili ba\u015fka bir \u00e7al\u0131\u015fmaya kat\u0131lan hastalarda intraven\u00f6z (IV) WVE-N531&#039;in potansiyel ke\u015fifsel biyobelirte\u00e7leri arac\u0131l\u0131\u011f\u0131yla uzun vadeli g\u00fcvenli\u011fini, tolere edilebilirli\u011fini, etkinli\u011fini, farmakokineti\u011fini ve farmakodinami\u011fini (PD) de\u011ferlendirmek i\u00e7in y\u00fcr\u00fct\u00fclen bir Faz 2 a\u00e7\u0131k etiketli uzatma (OLE) \u00e7al\u0131\u015fmas\u0131d\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma Ba\u015flang\u0131c\u0131 (Tahmini)<\/strong>: 2025-11<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birincil Tamamlanma (Tahmini)<\/strong>: 2029-03<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fman\u0131n Tamamlanmas\u0131 (Tahmini)<\/strong>: 2029-03<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Kat\u0131l\u0131mc\u0131 Say\u0131s\u0131 (Tahmini)<\/strong>: 175<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fma T\u00fcr\u00fc<\/strong>: M\u00fcdahaleci<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Konum<\/strong>: \u00dcrd\u00fcn, Birle\u015fik Krall\u0131k<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00c7al\u0131\u015fmaya Uygun Ya\u015flar<\/strong>: \u00c7ocuk, Yeti\u015fkin, Ya\u015fl\u0131 Yeti\u015fkin<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Temel dahil etme ve hari\u00e7 tutma kriterleri. \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT07209332\" target=\"_blank\" rel=\"noreferrer noopener\">NCT07209332<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-to-participate-in-the-wve-n-531-clinical-trial\">WVE-N531 Klinik Denemesine Nas\u0131l Kat\u0131l\u0131rs\u0131n\u0131z?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">WVE-N531 ekzon atlama tedavisi faz 2 klinik \u00e7al\u0131\u015fmas\u0131na kat\u0131lmak i\u00e7in a\u015fa\u011f\u0131daki ileti\u015fim bilgilerini kullanabilirsiniz. <strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne (DMD) \u0130\u00e7in Klinik Ara\u015ft\u0131rmalara Nas\u0131l Kat\u0131labilirsiniz?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0130sim<\/strong>: Klinik Operasyonlar<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Telefon Numaras\u0131<\/strong>: 855-215-4687<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-posta<\/strong>: clinicaltrials@wavelifesci.com<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Wave Life Sciences Ltd. announced on its ClinicalTrials.gov website that a phase 2 clinical trial of WVE-N531 (Exon 53 Skipping Therapy) will begin in November 2025 with 175 participants in Jordan and the United Kingdom. What is WVE-N531 Exon Skipping Therapy WVE-N531 is an investigational exon skipping therapy being developed as a potential therapy for [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4769,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,242,155,154,328,422,153,327],"class_list":["post-4762","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-clinical-trials","tag-exon-53","tag-exon-53-skipping","tag-exon-53-skipping-therapy","tag-exon-53-treatment","tag-wave-life-sciences","tag-wve-n531"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4762","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=4762"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4762\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4769"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4762"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4762"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4762"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}