{"id":8834,"date":"2026-10-01T15:39:32","date_gmt":"2026-10-01T12:39:32","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8834"},"modified":"2026-10-01T15:39:36","modified_gmt":"2026-10-01T12:39:36","slug":"brogidirsen-5-year-dmd-clinical-data","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/brogidirsen-5-year-dmd-clinical-data\/","title":{"rendered":"NS Pharma'n\u0131n Brogidirsen'i, Ekzon 44 Atlamaya Uygun DMD Hastalar\u0131nda 5 Y\u0131ll\u0131k Klinik Verileri Sunuyor"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>NS Pharma'n\u0131n Brogidirsen'i (NS-089\/NCNP-02), ekzon 44 atlamaya uygun distrofin geni mutasyonlar\u0131na sahip hastalar i\u00e7in tasarlanm\u0131\u015f, Duchenne kas distrofisi (DMD) i\u00e7in deneysel bir tedavidir.<\/strong> Brogidirsen&#039;in klinik geli\u015ftirme s\u00fcrecinden elde edilen be\u015f y\u0131ll\u0131k etkinlik ve g\u00fcvenlik verileri, 29 Eyl\u00fcl - 3 Ekim 2026 tarihleri aras\u0131nda Japonya&#039;n\u0131n Hiro\u015fima kentinde d\u00fczenlenen D\u00fcnya Kas Toplulu\u011fu&#039;nun 31. y\u0131ll\u0131k Uluslararas\u0131 Kongresi&#039;nde sunuldu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Merkezi Paramus, New Jersey'de bulunan NS Pharma, Inc., nadir hastal\u0131klar \u00fczerine odaklanm\u0131\u015f bir biyofarmas\u00f6tik \u015firketidir ve merkezi Kyoto, Japonya'da bulunan Nippon Shinyaku Co., Ltd.'nin bir i\u015ftirakidir. Brogidirsen, genetik mutasyonlar\u0131 ekzon 44 atlamas\u0131na uygun olan DMD hastalar\u0131 i\u00e7in deneysel bir tedavi olarak Nippon Shinyaku ve Ulusal N\u00f6roloji ve Psikiyatri Merkezi (NCNP) taraf\u0131ndan ortakla\u015fa ke\u015ffedilmi\u015ftir. <strong>Daha fazla bilgi edin<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noopener\">Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Yeni sunulan sonu\u00e7lar, NCNP taraf\u0131ndan y\u00fcr\u00fct\u00fclen ara\u015ft\u0131rmac\u0131 taraf\u0131ndan ba\u015flat\u0131lan bir klinik \u00e7al\u0131\u015fmadan ve Nippon Shinyaku taraf\u0131ndan y\u00fcr\u00fct\u00fclen a\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131ndan elde edilmi\u015ftir. \u00c7al\u0131\u015fmalar, haftal\u0131k intraven\u00f6z (IV) dozlama alan alt\u0131 kat\u0131l\u0131mc\u0131da Brogidirsen'in etkinli\u011fini ve g\u00fcvenli\u011fini de\u011ferlendirmi\u015ftir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Motor \u0130\u015flev Be\u015f Y\u0131l Boyunca Korundu<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fman\u0131n en \u00f6nemli bulgular\u0131ndan biri, \u00e7al\u0131\u015fma s\u00fcresince hareket kabiliyetini koruyan kat\u0131l\u0131mc\u0131lar aras\u0131nda motor fonksiyonun korunmas\u0131yd\u0131. De\u011ferlendirme, bu kat\u0131l\u0131mc\u0131larda motor fonksiyonun korundu\u011funu veya iyile\u015ftirildi\u011fini g\u00f6sterdi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma s\u00fcresince y\u00fcr\u00fcyemez hale gelenler de dahil olmak \u00fczere t\u00fcm kat\u0131l\u0131mc\u0131larda \u00fcst ekstremite fonksiyonu korunmu\u015ftur. Bu bulgular, uzun s\u00fcreli Brogidirsen tedavisi alan kat\u0131l\u0131mc\u0131lar\u0131n be\u015f y\u0131ll\u0131k de\u011ferlendirmesinin bir par\u00e7as\u0131yd\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sonu\u00e7lar, uzun s\u00fcreli tedavi boyunca fonksiyonel sonu\u00e7lara ili\u015fkin klinik veriler sa\u011flamakta ve brogidirsen'in, ekzon 44 atlamaya uygun DMD hastalar\u0131nda hastal\u0131k ilerlemesini yava\u015flatma potansiyeline sahip oldu\u011funu g\u00f6stermektedir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Tedaviyle \u0130li\u015fkili Ciddi veya \u015eiddetli Yan Etki Bildirilmemi\u015ftir<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Sunumun bir di\u011fer \u00f6nemli b\u00f6l\u00fcm\u00fc de be\u015f y\u0131ll\u0131k g\u00fcvenlik bulgular\u0131yd\u0131. Be\u015f y\u0131l boyunca brogidirsen alan kat\u0131l\u0131mc\u0131lar, uzun s\u00fcreli brogidirsen uygulamas\u0131na ba\u011fl\u0131 ciddi veya \u015fiddetli yan etkiler ya\u015famad\u0131lar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fmada ayr\u0131ca tedaviyle ili\u015fkili anafilaksi vakas\u0131 bildirilmedi ve be\u015f y\u0131ll\u0131k s\u00fcre boyunca hi\u00e7bir tedavi sonland\u0131r\u0131lmad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sunulan verilere g\u00f6re, bu bulgular, \u00e7al\u0131\u015fmada de\u011ferlendirilen kat\u0131l\u0131mc\u0131lar aras\u0131nda uzun s\u00fcreli uygulama i\u00e7in kabul edilebilir bir g\u00fcvenlik profiline i\u015faret etmektedir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DMD Do\u011fal Seyri ile Kar\u015f\u0131la\u015ft\u0131rma<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar ayr\u0131ca \u00e7e\u015fitli fonksiyonel \u00f6l\u00e7\u00fcmleri DMD&#039;nin do\u011fal seyri verileriyle kar\u015f\u0131la\u015ft\u0131rd\u0131lar. Bir\u00e7ok \u00f6l\u00e7\u00fcmde, \u00e7al\u0131\u015fmada g\u00f6zlemlenen fonksiyonel sonu\u00e7lar, hastal\u0131\u011f\u0131n do\u011fal seyriyle olumlu bir \u015fekilde kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, bulgular\u0131n Brogidirsen'in, mutasyonlar\u0131 ekzon 44 atlamas\u0131na uygun olan DMD hastalar\u0131nda hastal\u0131k ilerlemesini yava\u015flatma potansiyeline sahip olabilece\u011fini g\u00f6sterdi\u011fini belirtti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ancak, uzatma denemesi hala devam etmektedir. \u00c7al\u0131\u015fma, uzun s\u00fcreli brogidirsen uygulamas\u0131n\u0131n etkinli\u011fini ve g\u00fcvenli\u011fini ara\u015ft\u0131rmaya devam edecektir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">K\u00fcresel Faz II \u00c7al\u0131\u015fmas\u0131 Devam Ediyor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Devam eden geni\u015fletme \u00e7al\u0131\u015fmas\u0131na ek olarak, Nippon Shinyaku ve i\u015ftiraki NS Pharma, Brogidirsen'in k\u00fcresel bir Faz 2 \u00e7al\u0131\u015fmas\u0131n\u0131 y\u00fcr\u00fctmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">D\u00fcnya Kas Toplulu\u011fu Kongresi'nde sunulan be\u015f y\u0131ll\u0131k veriler, haftal\u0131k intraven\u00f6z Brogidirsen ile tedavi edilen alt\u0131 kat\u0131l\u0131mc\u0131dan elde edilen uzun vadeli klinik bilgileri i\u00e7ermektedir. Sonu\u00e7lar, motor fonksiyon, \u00fcst ekstremite fonksiyonu, g\u00fcvenlik ve DMD'nin do\u011fal seyri verileriyle kar\u015f\u0131la\u015ft\u0131rmalar\u0131 kapsamaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Brogidirsen halen ara\u015ft\u0131rma a\u015famas\u0131nda olan bir tedavi y\u00f6ntemidir ve devam eden \u00e7al\u0131\u015fmalar, ekzon 44 atlamaya uygun olan DMD hastalar\u0131nda etkinli\u011fi ve g\u00fcvenli\u011fi hakk\u0131nda ek bilgiler sa\u011flayacakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">Duchenne Klinik Ara\u015ft\u0131rma Yerleri Haritas\u0131<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>NS Pharma\u2019s brogidirsen (NS-089\/NCNP-02) is an investigational therapy for Duchenne muscular dystrophy (DMD) designed for patients with dystrophin gene mutations amenable to exon 44 skipping. Five-year efficacy and safety data from the clinical development of brogidirsen were presented at the 31st annual International Congress of the World Muscle Society, held in Hiroshima, Japan, from September [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":8835,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[138,137,141,127,246,136,924,140],"class_list":["post-8834","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-brogidirsen","tag-clinical-trial","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-nippon-shinyaku","tag-ns-pharma","tag-ns-089-ncnp-02"],"subtitle":"Brogidirsen, Duchenne kas distrofisi ara\u015ft\u0131rmalar\u0131nda dikkat \u00e7ekiyor. Yeni 5 y\u0131ll\u0131k veriler; motor fonksiyon ve uzun vadeli g\u00fcvenlilik konusundaki umut verici bulgularla birlikte, ilac\u0131n ekzon 44 atlamas\u0131na uygun hastalardaki potansiyelini ortaya koyuyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8834","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\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8834"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8834\/revisions"}],"predecessor-version":[{"id":8837,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8834\/revisions\/8837"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8835"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8834"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8834"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8834"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}