{"id":8726,"date":"2026-09-09T14:19:54","date_gmt":"2026-09-09T11:19:54","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8726"},"modified":"2026-09-09T14:19:57","modified_gmt":"2026-09-09T11:19:57","slug":"sat-3247-renamed-forazapadin-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/sat-3247-renamed-forazapadin-dmd\/","title":{"rendered":"Satellos&#039; Duchenne \u0130lac\u0131 SAT-3247, D\u00fcnya Sa\u011fl\u0131k \u00d6rg\u00fct\u00fc&#039;nden Resmi Ad\u0131n\u0131 Ald\u0131: Forazapadin"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Satellos Bioscience, ara\u015ft\u0131rma a\u015famas\u0131ndaki Duchenne kas distrofisi (DMD) tedavisi SAT-3247&#039;nin D\u00fcnya Sa\u011fl\u0131k \u00d6rg\u00fct\u00fc&#039;nden (WHO) resmi olarak Uluslararas\u0131 Tescilsiz \u0130sim (INN) Forazapadin ad\u0131n\u0131 ald\u0131\u011f\u0131n\u0131 duyurdu.<\/strong> \u0130lac\u0131n isimlendirilmesi, ilac\u0131n k\u00fcresel klinik geli\u015fimi ve gelecekteki d\u00fczenleyici s\u00fcreci i\u00e7in \u00f6nemli bir ad\u0131md\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-forazapadin\">Forazapadin nedir?<\/a><\/li><li><a href=\"#why-the-who-name-matters\">DS\u00d6 isminin \u00f6nemi neden b\u00fcy\u00fck?<\/a><\/li><li><a href=\"#phase-2-clinical-trials-continue\">2. Faz Klinik \u00c7al\u0131\u015fmalar\u0131 Devam Ediyor<\/a><\/li><li><a href=\"#why-this-could-be-important-for-the-dmd-community\">Bu, DMD toplulu\u011fu i\u00e7in neden \u00f6nemli olabilir?<\/a><\/li><li><a href=\"#dmd-warriors-take\">DMDWarrior&#039;\u0131n G\u00f6r\u00fc\u015f\u00fc<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 id=\"what-is-forazapadin\" class=\"wp-block-heading\">Forazapadin nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Forazapadin, Duchenne kas distrofisi olan ki\u015filerde kas yenilenmesini iyile\u015ftirmek i\u00e7in geli\u015ftirilen, a\u011f\u0131zdan al\u0131nan k\u00fc\u00e7\u00fck molek\u00fcll\u00fc bir ila\u00e7t\u0131r.<\/strong> Gen terapileri veya ekzon atlama tedavilerinin aksine, Forazapadin distrofin ba\u011f\u0131ms\u0131z bir mekanizma arac\u0131l\u0131\u011f\u0131yla \u00e7al\u0131\u015f\u0131r; yani hastan\u0131n ekzon mutasyonundan veya genetik alt tipinden ba\u011f\u0131ms\u0131z olarak kullan\u0131lmak \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu ila\u00e7, v\u00fccudun do\u011fal kas onar\u0131m s\u00fcrecinde rol oynayan AAK1 adl\u0131 bir proteini hedef al\u0131yor. Satellos, AAK1&#039;i bloke etmenin Duchenne kas distrofisinde bozulan kas yenilenmesini geri kazand\u0131rmaya yard\u0131mc\u0131 olabilece\u011fine inan\u0131yor.<\/p>\n\n\n\n<h2 id=\"why-the-who-name-matters\" class=\"wp-block-heading\">DS\u00d6 isminin \u00f6nemi neden b\u00fcy\u00fck?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Uluslararas\u0131 Tescilsiz \u0130sim (INN), DS\u00d6 adland\u0131rma s\u00fcreciyle verilen resmi jenerik isimdir. INN almak, bir tedavinin daha ileri bir geli\u015ftirme a\u015famas\u0131na ula\u015ft\u0131\u011f\u0131n\u0131 g\u00f6sterir ve bilimsel yay\u0131nlarda, klinik \u00e7al\u0131\u015fmalarda, d\u00fczenleyici ba\u015fvurularda ve gelecekteki ticarile\u015ftirmede tutarl\u0131 bir \u015fekilde kullan\u0131labilen k\u00fcresel olarak tan\u0131nan bir isim sa\u011flar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Satellos, bundan sonraki s\u00fcre\u00e7te bilimsel ve d\u00fczenleyici ileti\u015fimlerinde SAT-3247 ad\u0131n\u0131 kademeli olarak Forazapadin ile de\u011fi\u015ftirece\u011fini belirtiyor.<\/p>\n\n\n\n<h2 id=\"phase-2-clinical-trials-continue\" class=\"wp-block-heading\">2. Faz Klinik \u00c7al\u0131\u015fmalar\u0131 Devam Ediyor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Forazapadin \u015fu anda Duchenne kas distrofisi i\u00e7in iki Faz 2 klinik \u00e7al\u0131\u015fmada de\u011ferlendirilmektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>BASECAMP<\/strong> \u2014 DMD&#039;li \u00e7ocuklarda yap\u0131lan rastgele, plasebo kontroll\u00fc k\u00fcresel bir \u00e7al\u0131\u015fma.<\/li>\n\n\n\n<li><strong>TRAILHEAD<\/strong> \u2014 DMD&#039;li yeti\u015fkinlerde yap\u0131lan a\u00e7\u0131k etiketli Faz 2 \u00e7al\u0131\u015fmas\u0131.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Satellos&#039;ye g\u00f6re, BASECAMP \u00e7al\u0131\u015fmas\u0131na kay\u0131tlar\u0131n bu \u00e7eyrekte tamamlanmas\u0131 beklenirken, \u00e7al\u0131\u015fmadan elde edilecek ilk klinik verilerin 2026&#039;n\u0131n d\u00f6rd\u00fcnc\u00fc \u00e7eyre\u011finde a\u00e7\u0131klanmas\u0131 \u00f6ng\u00f6r\u00fcl\u00fcyor. Daha fazla bilgi edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/clinical-trials\/\" target=\"_blank\" rel=\"noopener\">Duchenne Klinik Ara\u015ft\u0131rmalar\u0131n\u0131 Ke\u015ffedin<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket ayr\u0131ca, 2026 y\u0131l\u0131n\u0131n sonundan \u00f6nce fasyoskapulohumeral kas distrofisi (FSHD) hastal\u0131\u011f\u0131nda Forazapadin&#039;in Faz 2 klinik denemesine ba\u015flamay\u0131 planl\u0131yor.<\/p>\n\n\n\n<h2 id=\"why-this-could-be-important-for-the-dmd-community\" class=\"wp-block-heading\">Bu, DMD toplulu\u011fu i\u00e7in neden \u00f6nemli olabilir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">G\u00fcn\u00fcm\u00fczdeki DMD tedavilerinin \u00e7o\u011fu belirli genetik mutasyonlar\u0131 hedef al\u0131yor veya distrofin \u00fcretimini eski haline getirmeye odaklan\u0131yor. Forazapadin ise farkl\u0131 bir ama\u00e7la geli\u015ftiriliyor: distrofin \u00fcretiminden ba\u011f\u0131ms\u0131z olarak, v\u00fccudun iskelet kas\u0131n\u0131 yenileme yetene\u011fini desteklemek.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik denemelerde ba\u015far\u0131l\u0131 olmas\u0131 durumunda, bu yakla\u015f\u0131m potansiyel olarak \u015fu ama\u00e7larla kullan\u0131labilir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Farkl\u0131 Duchenne mutasyon tiplerinde.<\/li>\n\n\n\n<li>Di\u011fer onaylanm\u0131\u015f DMD tedavilerinin yan\u0131 s\u0131ra.<\/li>\n\n\n\n<li>Kas sa\u011fl\u0131\u011f\u0131n\u0131 korumay\u0131 ama\u00e7layan ba\u011f\u0131ms\u0131z bir tedavi y\u00f6ntemi olarak.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Ancak, Forazapadin&#039;in halen ara\u015ft\u0131rma a\u015famas\u0131nda olan bir ila\u00e7 oldu\u011funu belirtmek \u00f6nemlidir. G\u00fcvenli\u011fi ve etkinli\u011fi devam eden klinik \u00e7al\u0131\u015fmalarda de\u011ferlendirilmekte olup, Duchenne kas distrofisi i\u00e7in hen\u00fcz ruhsat onay\u0131 almam\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 id=\"dmd-warriors-take\" class=\"wp-block-heading\">DMDWarrior&#039;\u0131n G\u00f6r\u00fc\u015f\u00fc<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Forazapadin ad\u0131n\u0131n verilmesi bir onay anlam\u0131na gelmemekle birlikte, Satellos'un Duchenne geli\u015ftirme program\u0131n\u0131n olgunlu\u011funu yans\u0131tan anlaml\u0131 bir kilometre ta\u015f\u0131d\u0131r. DMDWarrior, BASECAMP ve TRAILHEAD klinik \u00e7al\u0131\u015fmalar\u0131n\u0131 yak\u0131ndan takip etmeye ve yeni etkinlik, g\u00fcvenlik ve biyomarker g\u00fcncellemeleri yay\u0131nland\u0131k\u00e7a bunlar\u0131 bildirmeye devam edecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong><a href=\"https:\/\/dmdwarrior.com\/tr\/duchenne-exon-deletion-tool\/\" target=\"_blank\" data-type=\"link\" data-id=\"https:\/\/dmdwarrior.com\/duchenne-exon-deletion-tool\/\" rel=\"noreferrer noopener\">Forazapadin'e uygun musunuz?<\/a><\/strong> Hemen kontrol edin!<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Satellos Bioscience has announced that its investigational Duchenne muscular dystrophy (DMD) treatment SAT-3247 has officially received the International Nonproprietary Name (INN) Forazapadin from the World Health Organization (WHO). The naming milestone is an important step in the drug\u2019s global clinical development and future regulatory pathway. What is Forazapadin? Forazapadin is an oral small-molecule medicine being [&hellip;]<\/p>\n","protected":false},"author":8,"featured_media":8729,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[903,251,252,253],"class_list":["post-8726","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-forazapadin","tag-sat-3247","tag-satellos","tag-satellos-bioscience"],"subtitle":"Duchenne kas distrofisi ilac\u0131 SAT-3247'nin yeni ad\u0131 Forazapadin oldu. D\u00fcnya Sa\u011fl\u0131k \u00d6rg\u00fct\u00fc'n\u00fcn bu onay\u0131, ilac\u0131n geli\u015ftirilmesinde \u00f6nemli bir ad\u0131m\u0131 i\u015faret ediyor. Peki bu yeni oral tedavi, DMD tedavisinin gelece\u011fi i\u00e7in ne anlama geliyor?","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8726","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\/8"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8726"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8726\/revisions"}],"predecessor-version":[{"id":8730,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8726\/revisions\/8730"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8729"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8726"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8726"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8726"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}