{"id":4799,"date":"2025-10-14T10:10:06","date_gmt":"2025-10-14T07:10:06","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4799"},"modified":"2025-10-14T10:17:10","modified_gmt":"2025-10-14T07:17:10","slug":"brogidirsen-ns-089-ncnp-02-has-the-potential-to-slow-disease-progression-in-dmd-patients-amenable-to-exon-44-skipping","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/brogidirsen-ns-089-ncnp-02-has-the-potential-to-slow-disease-progression-in-dmd-patients-amenable-to-exon-44-skipping\/","title":{"rendered":"Brogidirsen (NS-089\/NCNP-02), Ekzon 44 Atlamaya Uygun DMD Hastalar\u0131nda, Hastal\u0131\u011f\u0131n \u0130lerlemesini Yava\u015flatma Potansiyeline Sahip"},"content":{"rendered":"

Nippon Shinyaku, brogidirsen (NS-089\/NCNP-02) i\u00e7in klinik bir \u00e7al\u0131\u015fman\u0131n ba\u015flat\u0131lmas\u0131n\u0131 da i\u00e7eren a\u00e7\u0131k etiketli bir uzatma \u00e7al\u0131\u015fmas\u0131na dayanan 3,5 y\u0131ll\u0131k bir ba\u015fvurudan elde edilen etkililik ve g\u00fcvenlik verilerini duyurdu.<\/p>\n\n\n\n

Bu veriler, 7-11 Ekim 2025 tarihleri aras\u0131nda Avusturya'da d\u00fczenlenen 30. D\u00fcnya Kas Toplulu\u011fu Uluslararas\u0131 Kongresi'nde Ulusal N\u00f6roloji ve Psikiyatri Merkezi (NCNP, Kodaira \u015eehri, Tokyo; Ba\u015fkan, Kazuyuki Nakagome) taraf\u0131ndan poster olarak sunuldu.<\/p>\n\n\n\n

Brogidirsen nedir?<\/h2>\n\n\n\n

Brogidirsen, Nippon Shinyaku ve NCNP taraf\u0131ndan ekzon 44 atlamaya uygun distrofin gen mutasyonlar\u0131na sahip DMD hastalar\u0131 i\u00e7in deneysel bir tedavi olarak ortak ke\u015ffedilen bir antisens oligon\u00fckleotiddir.<\/p>\n\n\n\n

Sunulan veriler, NCNP taraf\u0131ndan y\u00fcr\u00fct\u00fclen ara\u015ft\u0131rmac\u0131 taraf\u0131ndan ba\u015flat\u0131lan klinik \u00e7al\u0131\u015fmaya ve Nippon Shinyaku taraf\u0131ndan y\u00fcr\u00fct\u00fclen uzatma \u00e7al\u0131\u015fmas\u0131na dayanmaktad\u0131r. Bu \u00e7al\u0131\u015fmalarda, brogidirsen'in haftal\u0131k intraven\u00f6z uygulamas\u0131n\u0131 alan 6 hastada etkinlik ve g\u00fcvenlik de\u011ferlendirilmi\u015ftir.<\/p>\n\n\n\n

Devam\u0131n\u0131 oku<\/strong>: Yakla\u015fan Exon 44 Atlama Terapileri<\/a><\/p>\n\n\n\n

G\u00fcvenlik Sonu\u00e7lar\u0131<\/h2>\n\n\n\n

Sonu\u00e7lar, 25\/26. haftalarda ve 99\/100. haftalarda biyopsi yap\u0131lan kaslarda y\u00fcksek ekzon atlama etkinli\u011fi ve distrofin ekspresyon d\u00fczeyi e\u011filimleri g\u00f6sterdi. Ayr\u0131ca, uzun s\u00fcreli brogidirsen uygulamas\u0131ndan sonra hareketsiz kalan kat\u0131l\u0131mc\u0131lar, North Star Ambulatuvar De\u011ferlendirmesi de dahil olmak \u00fczere de\u011ferlendirmelerde motor fonksiyon yeteneklerini korudular. G\u00fcvenlik de\u011ferlendirme sonu\u00e7lar\u0131, brogidirsen ile ili\u015fkili ciddi veya \u015fiddetli advers olay veya anafilaksi gibi inf\u00fczyonla ili\u015fkili reaksiyonlarla ili\u015fkili advers olay veya advers olaylar nedeniyle tedavinin kesilmesi g\u00f6zlemlenmedi\u011fini g\u00f6sterdi.<\/p>\n\n\n\n

Sonu\u00e7lar, brogidirsen'in ekzon 44 atlamaya uygun DMD'li hastalarda hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatma potansiyeline sahip oldu\u011funu g\u00f6stermektedir.<\/strong><\/p>\n\n\n\n

Bu uzun vadeli uzatma \u00e7al\u0131\u015fmas\u0131, uzun vadeli uygulaman\u0131n etkinli\u011fini ve g\u00fcvenli\u011fini ara\u015ft\u0131rmak amac\u0131yla devam etmektedir. Ayr\u0131ca, brogidirsen ile ilgili k\u00fcresel bir Faz II \u00e7al\u0131\u015fmas\u0131, Nippon Shinyaku ve ba\u011fl\u0131 kurulu\u015fu NS Pharma, Inc. taraf\u0131ndan y\u00fcr\u00fct\u00fclmektedir.<\/p>\n\n\n\n

Daha fazla bilgi edin<\/strong>:\u00a0Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an open-label extension study that also included the initiation of a clinical trial for brogidirsen (NS-089\/NCNP-02). These data were presented as a poster by the National Center of Neurology and Psychiatry (NCNP, Kodaira City, Tokyo; President, Kazuyuki Nakagome) at the 30th annual […]<\/p>\n","protected":false},"author":1,"featured_media":4800,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[138,141,127,246,136,140],"class_list":["post-4799","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-brogidirsen","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-nippon-shinyaku","tag-ns-089-ncnp-02"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4799","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=4799"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4799\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4800"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4799"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4799"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4799"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}