{"id":7866,"date":"2026-05-07T14:46:47","date_gmt":"2026-05-07T11:46:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7866"},"modified":"2026-05-07T16:36:59","modified_gmt":"2026-05-07T13:36:59","slug":"entrada-therapeutics-phase-1-2-results-for-entr-601-44-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/entrada-therapeutics-phase-1-2-results-for-entr-601-44-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Entrada Therapeutics, Duchenne Musk\u00fcler Distrofisinde ENTR-601-44 \u0130\u00e7in Faz 1\/2 Sonu\u00e7lar\u0131n\u0131 A\u00e7\u0131klad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Entrada Therapeutics, Duchenne Musk\u00fcler Distrofisi (DMD) hastalar\u0131 i\u00e7in deneysel bir tedavi olan ENTR-601-44'nin erken klinik deneme sonu\u00e7lar\u0131n\u0131 a\u00e7\u0131klad\u0131. Bu terapi, v\u00fccudun daha fazla distrofin proteini \u00fcretmesine yard\u0131mc\u0131 olmay\u0131 ama\u00e7layan genetik bir yakla\u015f\u0131m olan \"ekzon 44 atlama\"dan fayda g\u00f6rebilecek hastalar i\u00e7in tasarlanm\u0131\u015ft\u0131r. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ancak Entrada Therapeutics yeni nesil Duchenne tedavisi hayal k\u0131r\u0131kl\u0131\u011f\u0131 yaratan sonu\u00e7lar verdi.<\/strong> Verilerin a\u00e7\u0131klanmas\u0131n\u0131n ard\u0131ndan, Duchenne kas distrofisi olan \u00e7ocuklar i\u00e7in geli\u015ftirilen ila\u00e7, erken ve orta a\u015fama klinik denemelerinde analistlerin beklentilerinin \u00f6nemli \u00f6l\u00e7\u00fcde alt\u0131nda kald\u0131 ve bu durum \u015firketin hisselerinin Per\u015fembe g\u00fcn\u00fc piyasa \u00f6ncesi i\u015flemlerde y\u00fczde 50'den fazla d\u00fc\u015fmesine neden oldu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Distrofin, kaslar\u0131n g\u00fc\u00e7l\u00fc kalmas\u0131na yard\u0131mc\u0131 olan \u00f6nemli bir proteindir. DMD hastalar\u0131nda distrofin seviyesi \u00e7ok d\u00fc\u015f\u00fckt\u00fcr veya hi\u00e7 yoktur, bu da ilerleyici kas g\u00fc\u00e7s\u00fczl\u00fc\u011f\u00fcne yol a\u00e7ar. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/dystrophin-gene\/\" target=\"_blank\" data-type=\"post\" data-id=\"3785\" rel=\"noreferrer noopener\">Distrofin Geni<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\">\u00c7al\u0131\u015fmada Neler Oldu?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Faz 1\/2 ELEVATE-44-201 \u00e7al\u0131\u015fmas\u0131na 4 ila 20 ya\u015f aras\u0131 y\u00fcr\u00fcyebilen erkek \u00e7ocuklar ve gen\u00e7 erkekler dahil edildi. Kat\u0131l\u0131mc\u0131lara ya ENTR-601-44 ya da plasebo verildi. Ara\u015ft\u0131rmac\u0131lar esas olarak g\u00fcvenli\u011fi, distrofin \u00fcretimini ve fiziksel i\u015flevi de\u011ferlendirdi.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Ana Bulgular<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar \u015funlar\u0131 bildirdi:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Entrada Therapeutics, ekzon 44 atlamas\u0131na uygun mutasyonlardan kaynaklanan Duchenne Musk\u00fcler Distrofisi ile ya\u015fayan, 6 ila 17 ya\u015f aras\u0131 ayakta durabilen erkek \u00e7ocuklarda ENTR-601-44'yi de\u011ferlendirdi.<\/li>\n\n\n\n<li><strong>Boston merkezli biyoteknoloji \u015firketi, tedavi g\u00f6ren hastalarda distrofin ekspresyonunda 4% bazal seviyesine g\u00f6re %2,36&#039;l\u0131k bir art\u0131\u015f sa\u011fland\u0131\u011f\u0131n\u0131 ve Duchenne hastalar\u0131nda eksik olan kritik kas proteininin bir k\u0131sm\u0131n\u0131n geri kazand\u0131r\u0131ld\u0131\u011f\u0131n\u0131 bildirdi.<\/strong><\/li>\n\n\n\n<li>William Blair analisti Myles Minter, Nisan ay\u0131nda yay\u0131nlanan bir ara\u015ft\u0131rma notunda, plasebo ile d\u00fczeltilmi\u015f distrofin art\u0131\u015f\u0131n\u0131n en az 10% olmas\u0131n\u0131n, tedavi i\u00e7in beklenen temel senaryoyu temsil etti\u011fini, firman\u0131n i\u00e7 projeksiyonlar\u0131n\u0131n ise yakla\u015f\u0131k 11% olarak tahmin etti\u011fini belirtti.<\/li>\n\n\n\n<li><strong>Bulgular ayr\u0131ca, daha \u00f6nce orta a\u015fama bir klinik \u00e7al\u0131\u015fmada plasebo ile ayarlanm\u0131\u015f distrofin kazan\u0131mlar\u0131nda yakla\u015f\u0131k 25%'lik bir art\u0131\u015f g\u00f6steren ve Oppenheimer analistlerinin Entrada'nin program\u0131 i\u00e7in ba\u015far\u0131 \u00f6l\u00e7\u00fct\u00fc olarak tan\u0131mlad\u0131\u011f\u0131 del-zota, Avidity Biosciences' rakip ekzon 44 atlama aday\u0131 ile de olumsuz bir \u015fekilde kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131.<\/strong><\/li>\n\n\n\n<li>Verilerin yay\u0131nlanmas\u0131ndan \u00f6nce Minter, ENTR-601-44 i\u00e7in en b\u00fcy\u00fck endi\u015fe kayna\u011f\u0131n\u0131n kaslara ila\u00e7 iletim verimlili\u011fi oldu\u011funu belirtmi\u015fti ve Entrada daha sonra pediatrik hastalarda sistemik ila\u00e7 maruziyetinin yeti\u015fkin kat\u0131l\u0131mc\u0131lara k\u0131yasla beklenenden daha d\u00fc\u015f\u00fck oldu\u011funu a\u00e7\u0131klam\u0131\u015ft\u0131.<\/li>\n\n\n\n<li><strong>Duchenne kas distrofisi, \u00e7o\u011funlukla erkek \u00e7ocuklar\u0131 etkileyen ve zamanla ilerleyici kas dejenerasyonuna yol a\u00e7an nadir g\u00f6r\u00fclen kal\u0131tsal bir n\u00f6rom\u00fcsk\u00fcler hastal\u0131kt\u0131r.<\/strong><\/li>\n\n\n\n<li>\u00c7al\u0131\u015fma ayr\u0131ca, tedavi g\u00f6ren kat\u0131l\u0131mc\u0131lar\u0131n yerden aya\u011fa kalkmalar\u0131 i\u00e7in gereken s\u00fcrede istatistiksel olarak anlaml\u0131 bir iyile\u015fme oldu\u011funu g\u00f6sterdi; bu s\u00fcre, kas g\u00fcc\u00fcn\u00fc izlemek ve y\u00fcr\u00fcme yetene\u011finin kayb\u0131n\u0131 tahmin etmek i\u00e7in yayg\u0131n olarak kullan\u0131lan bir fonksiyonel de\u011ferlendirme y\u00f6ntemidir.<\/li>\n\n\n\n<li><strong>Entrada ayr\u0131ca, orijinal dozun iki kat\u0131n\u0131 alan ikinci bir hasta grubunda dozlaman\u0131n ba\u015flad\u0131\u011f\u0131n\u0131 ve ek klinik sonu\u00e7lar\u0131n 2026 y\u0131l\u0131n\u0131n sonundan \u00f6nce beklendi\u011fini do\u011frulad\u0131.<\/strong><\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\">Bu terapiyi farkl\u0131 k\u0131lan nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Entrada bilim insanlar\u0131, geli\u015ftirdikleri tedavi teknolojisinin, uydu h\u00fccreleri ad\u0131 verilen kas k\u00f6k h\u00fccrelerine ula\u015fmas\u0131na yard\u0131mc\u0131 olabilece\u011fine inan\u0131yor. Bu h\u00fccreler, hasar g\u00f6rm\u00fc\u015f kaslar\u0131n onar\u0131lmas\u0131na ve yeni kas liflerinin olu\u015fturulmas\u0131na yard\u0131mc\u0131 olduklar\u0131 i\u00e7in \u00f6nemlidir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar, distrofin art\u0131\u015flar\u0131 nispeten m\u00fctevaz\u0131 olsa bile baz\u0131 hastalar\u0131n erken d\u00f6nemde fonksiyonel iyile\u015fmeler ya\u015famalar\u0131n\u0131n nedeninin bu olabilece\u011fini d\u00fc\u015f\u00fcn\u00fcyor. <\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\">Bundan sonra ne olacak?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Birinci gruptaki t\u00fcm kat\u0131l\u0131mc\u0131lar art\u0131k \u00e7al\u0131\u015fman\u0131n a\u00e7\u0131k etiketli b\u00f6l\u00fcm\u00fcne ge\u00e7tiler ve burada herkes tedavi al\u0131yor. Ara\u015ft\u0131rmac\u0131lar ayr\u0131ca ek hasta gruplar\u0131nda daha y\u00fcksek dozlar\u0131 da test ediyorlar.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Entrada, 2026 y\u0131l\u0131n\u0131n ilerleyen d\u00f6nemlerinde daha fazla sonu\u00e7 bekliyor.<\/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>Entrada Therapeutics announced early clinical trial results for ENTR-601-44, an experimental treatment for people with Duchenne Muscular Dystrophy (DMD). The therapy is designed for patients who can benefit from \u201cexon 44 skipping,\u201d a genetic approach aimed at helping the body produce more dystrophin protein. Learn More: Mutations and Deletions Amenable to Exon 44 Skipping Therapies [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7867,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[137,265,233,234,141,127,246],"class_list":["post-7866","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trial","tag-entr-601-44","tag-entrada","tag-entrada-therapeutics","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7866","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=7866"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/7866\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/7867"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=7866"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=7866"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=7866"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}