{"id":6747,"date":"2026-03-12T11:43:48","date_gmt":"2026-03-12T08:43:48","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6747"},"modified":"2026-06-24T22:01:09","modified_gmt":"2026-06-24T19:01:09","slug":"del-zota-phase-3-safari44-clinical-trial-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/del-zota-phase-3-safari44-clinical-trial-duchenne-muscular-dystrophy\/","title":{"rendered":"Duchenne Kas Distrofisi i\u00e7in Del-Zota Faz 3 SAFARI44 Klinik \u00c7al\u0131\u015fmas\u0131: \u00c7al\u0131\u015fma Tasar\u0131m\u0131, Veriler ve Klinik \u00d6nemi"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Duchenne kas distrofisi i\u00e7in y\u00fcr\u00fct\u00fclen del-zota faz 3 SAFARI44 klinik \u00e7al\u0131\u015fmas\u0131, Duchenne kas distrofisinde ekzon 44 atlamaya uygun genetik mutasyonlara sahip hastalar i\u00e7in hedefe y\u00f6nelik tedaviler geli\u015ftirmeye y\u00f6nelik \u00f6nemli bir ad\u0131m\u0131 temsil etmektedir. <\/strong>Daha fazla bilgi edin: <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\">Kas Distrofisi Derne\u011fi Klinik ve Bilimsel Konferans\u0131'nda sunulan SAFARI44 \u00e7al\u0131\u015fmas\u0131, Avidity Biosciences taraf\u0131ndan geli\u015ftirilen deneysel bir antikor-oligon\u00fckleotid konjugat\u0131 (AOC) tedavisi olan del-zota'n\u0131n g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendirmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Faz 3 klinik denemesi, del-zota'n\u0131n ekzon 44 mutasyonlar\u0131ndan kaynaklanan DMD hastalar\u0131nda distrofin \u00fcretimini ve kas fonksiyonunu \u00f6nemli \u00f6l\u00e7\u00fcde iyile\u015ftirebilece\u011fini g\u00f6steren cesaret verici erken a\u015fama verilerine dayanmaktad\u0131r.<\/strong><\/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=\"#key-highlights-of-the-safari-44-phase-3-trial\">SAFARI44 Faz 3 Klinik \u00c7al\u0131\u015fmas\u0131 Nedir?<\/a><\/li><li><a href=\"#primary-and-secondary-endpoints\">Birincil ve \u0130kincil Sonu\u00e7 Noktalar\u0131<\/a><\/li><li><a href=\"#mechanism-of-del-zota-therapy\">Del-Zota Tedavisinin Mekanizmas\u0131<\/a><\/li><li><a href=\"#earlier-clinical-data-explore-44-trial\">\u00d6nceki Klinik Veriler: EXPLORE44 Klinik \u00c7al\u0131\u015fmas\u0131<\/a><ul><li><a href=\"#key-findings-from-explore-44\">EXPLORE44&#039;den Elde Edilen Ba\u015fl\u0131ca Bulgular<\/a><\/li><\/ul><\/li><li><a href=\"#open-label-extension-ole-results\">A\u00e7\u0131k Etiketli Geni\u015fletme (OLE) Sonu\u00e7lar\u0131<\/a><\/li><li><a href=\"#safety-and-tolerability\">G\u00fcvenlik ve Tolerans<\/a><\/li><li><a href=\"#why-the-safari-44-trial-matters\">SAFARI44 Klinik Ara\u015ft\u0131rmas\u0131n\u0131n \u00d6nemi<\/a><\/li><li><a href=\"#in-which-countries-will-the-del-zota-phase-3-safari-44-clinical-trials-begin\">Del-Zota Faz 3 SAFARI44 Klinik \u00c7al\u0131\u015fmalar\u0131 hangi \u00fclkelerde ba\u015flayacak?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"key-highlights-of-the-safari-44-phase-3-trial\" class=\"wp-block-heading\">SAFARI44 Faz 3 Klinik \u00c7al\u0131\u015fmas\u0131 Nedir?<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Ya\u015flar\u0131 2 ve \u00fczeri olan yakla\u015f\u0131k 70 ayakta tedavi g\u00f6ren hasta kat\u0131lacakt\u0131r.<\/strong><\/li>\n\n\n\n<li>Kat\u0131l\u0131mc\u0131lara 54 hafta boyunca alt\u0131 haftada bir intraven\u00f6z dozlama yap\u0131lacakt\u0131r.<\/li>\n\n\n\n<li><strong>Klinik \u00e7al\u0131\u015fma \u015funlar\u0131 i\u00e7ermektedir:<\/strong>\n<ul class=\"wp-block-list\">\n<li>Rastgele \u00e7ift k\u00f6r tedavi a\u015famas\u0131 (54 hafta)<\/li>\n\n\n\n<li><strong>A\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131 (54 hafta)<\/strong><\/li>\n\n\n\n<li>G\u00fcvenlik takibi (6 hafta)<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>Kat\u0131l\u0131mc\u0131lar 1:1 oran\u0131nda rastgele da\u011f\u0131l\u0131mla del-zota 5 mg\/kg PMO (toplam AOC a\u011f\u0131rl\u0131\u011f\u0131 ba\u015f\u0131na 28 mg\/kg) veya plasebo alacaklard\u0131r.<\/strong><\/li>\n\n\n\n<li>K\u00f6rleme y\u00f6ntemiyle y\u00fcr\u00fct\u00fclen tedavi d\u00f6neminde toplam dokuz inf\u00fczyon uygulanacakt\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu tasar\u0131m, del-zota'n\u0131n Duchenne kas distrofisi i\u00e7in temel tedavi hedefleri olan kas fonksiyonunu ve distrofin \u00fcretimini \u00f6nemli \u00f6l\u00e7\u00fcde iyile\u015ftirip iyile\u015ftiremeyece\u011fini belirlemeyi ama\u00e7lamaktad\u0131r.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"primary-and-secondary-endpoints\" class=\"wp-block-heading\">Birincil ve \u0130kincil Sonu\u00e7 Noktalar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Del-Zota faz 3 SAFARI44 klinik \u00e7al\u0131\u015fmas\u0131n\u0131n birincil sonlanma noktas\u0131 \u015fudur:<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Y\u00fckselme s\u00fcresindeki h\u0131z de\u011fi\u015fimi<\/strong>: Alt ekstremite kas g\u00fcc\u00fcn\u00fc yans\u0131tan, yayg\u0131n olarak kullan\u0131lan fonksiyonel bir \u00f6l\u00e7\u00fcm.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0130kincil sonu\u00e7 \u00f6l\u00e7\u00fctleri \u015funlard\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Kreatin kinaz (CK) seviyeleri<\/strong><\/li>\n\n\n\n<li><strong>4 basamakl\u0131 merdiven \u00e7\u0131kma h\u0131z\u0131<\/strong><\/li>\n\n\n\n<li><strong>10 metrelik y\u00fcr\u00fcme\/ko\u015fma testi<\/strong><\/li>\n\n\n\n<li><strong>SV95C ad\u0131m h\u0131z\u0131<\/strong><\/li>\n\n\n\n<li><strong>Kuzey Y\u0131ld\u0131z\u0131 Ayaktan De\u011ferlendirme (NSAA)<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00f6l\u00e7\u00fctler, DMD klinik \u00e7al\u0131\u015fmalar\u0131nda hastal\u0131k ilerlemesini ve fonksiyonel iyile\u015fmeyi de\u011ferlendirmek i\u00e7in yayg\u0131n olarak kullan\u0131lmaktad\u0131r. Daha fazla bilgi edinin:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD&#039;de Biyobelirte\u00e7ler<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"mechanism-of-del-zota-therapy\" class=\"wp-block-heading\">Del-Zota Tedavisinin Mekanizmas\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Del-zota, fosforodiamidat morfolino oligomerlerini (PMO&#039;lar) do\u011frudan kas dokusuna iletmek \u00fczere tasarlanm\u0131\u015f bir antikor-oligon\u00fckleotid konjugat\u0131d\u0131r (AOC).<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu terapinin amac\u0131 \u015funlard\u0131r:<\/p>\n\n\n\n<ol class=\"wp-block-list\">\n<li><strong>Distrofin genindeki 44. ekzonu atlamak.<\/strong><\/li>\n\n\n\n<li><strong>Distrofin mRNA's\u0131n\u0131n okuma \u00e7er\u00e7evesini geri y\u00fcklemek.<\/strong><\/li>\n\n\n\n<li><strong>Daha k\u0131sa ama i\u015flevsel bir distrofin proteininin \u00fcretimini m\u00fcmk\u00fcn k\u0131lmak.<\/strong><\/li>\n<\/ol>\n\n\n\n<p class=\"wp-block-paragraph\">Bu strateji, mutasyonlar\u0131 ekzon atlama tedavilerine uygun olan hastalarda kas liflerini stabilize etmeye ve hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatmaya yard\u0131mc\u0131 olabilir.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"earlier-clinical-data-explore-44-trial\" class=\"wp-block-heading\">\u00d6nceki Klinik Veriler: EXPLORE44 Klinik \u00c7al\u0131\u015fmas\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>EXPLORE44 \u00e7al\u0131\u015fmas\u0131, ekzon 44 atlamaya uygun Duchenne kas distrofisi olan 26 kat\u0131l\u0131mc\u0131y\u0131 i\u00e7eren EXPLORE44 faz 1\/2 klinik \u00e7al\u0131\u015fmas\u0131n\u0131n sonu\u00e7lar\u0131na dayanmaktad\u0131r.<\/strong><\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img decoding=\"async\" width=\"968\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR.jpg\" alt=\"Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar\" class=\"wp-image-5275\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR.jpg 1890w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-284x300.jpg 284w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-968x1024.jpg 968w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-768x813.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-1452x1536.jpg 1452w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-11x12.jpg 11w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-397x420.jpg 397w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-150x159.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-300x317.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-696x737.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/12\/Deletions-Amenable-to-Exon-44-Skipping-TR-1068x1130.jpg 1068w\" sizes=\"(max-width: 968px) 100vw, 968px\" \/><\/figure>\n\n\n\n<h3 id=\"key-findings-from-explore-44\" class=\"wp-block-heading\">EXPLORE44&#039;den Elde Edilen Ba\u015fl\u0131ca Bulgular<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Klinik veriler \u015funu g\u00f6stermi\u015ftir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0130skelet kas\u0131nda ~200 nM PMO konsantrasyonlar\u0131<\/strong><\/li>\n\n\n\n<li><strong>~25% normal distrofin seviyeleri<\/strong><\/li>\n\n\n\n<li><strong>37% ekzon-44 atlamas\u0131nda art\u0131\u015f<\/strong><\/li>\n\n\n\n<li><strong>&gt;80% kreatin kinazda azalma<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Fonksiyonel sonu\u00e7lar da do\u011fal seyir g\u00f6steren kohortlarla kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda cesaret verici sonu\u00e7lar ortaya koydu. Daha Fazlas\u0131n\u0131 Okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping\/\" target=\"_blank\" rel=\"noreferrer noopener\">Del-zota (EXPLORE44), Normal Distrofin \u00dcretiminin Yakla\u015f\u0131k 30%&#039;sini Sa\u011flar<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"open-label-extension-ole-results\" class=\"wp-block-heading\">A\u00e7\u0131k Etiketli Geni\u015fletme (OLE) Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>A\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131na kat\u0131lan ve tedaviye devam eden kat\u0131l\u0131mc\u0131lar, 12-24 ay boyunca \u00f6l\u00e7\u00fclebilir fonksiyonel faydalar elde ettiler.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">G\u00f6zlemlenen sonu\u00e7lar \u015funlard\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>4 basamakl\u0131 merdiven \u00e7\u0131kma testi<\/strong>\n<ul class=\"wp-block-list\">\n<li>2,1 saniyelik iyile\u015fme<\/li>\n\n\n\n<li><strong>Do\u011fal tarih kohortunda 2,7 saniyelik bir d\u00fc\u015f\u00fc\u015f ya\u015fand\u0131.<\/strong><\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>10 metrelik y\u00fcr\u00fcy\u00fc\u015f\/ko\u015fu\n<ul class=\"wp-block-list\">\n<li><strong>Tedavi edilmeyen gruplarda 1,5 saniyelik d\u00fc\u015f\u00fc\u015fe kar\u015f\u0131l\u0131k 0,7 saniyelik iyile\u015fme.<\/strong><\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Yerden kalkma zaman\u0131\n<ul class=\"wp-block-list\">\n<li><strong>3,2 saniyelik iyile\u015fme, 1,6 saniyelik d\u00fc\u015f\u00fc\u015fe kar\u015f\u0131l\u0131k.<\/strong><\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Ek bulgular \u015funlar\u0131 i\u00e7eriyordu:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Tedavi g\u00f6ren hastalarda stabil NSAA skorlar\u0131<\/strong><\/li>\n\n\n\n<li><strong>\u00dcst Ekstremite Performans\u0131 (PUL) puanlar\u0131nda 1,5 puanl\u0131k iyile\u015fme<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu sonu\u00e7lar, del-zota'n\u0131n Duchenne kas distrofisi tedavisinde \u00f6nemli bir hedef olan fonksiyonel gerilemeyi yava\u015flatabilece\u011fini veya stabilize edebilece\u011fini d\u00fc\u015f\u00fcnd\u00fcrmektedir.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"safety-and-tolerability\" class=\"wp-block-heading\">G\u00fcvenlik ve Tolerans<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Erken d\u00f6nem klinik \u00e7al\u0131\u015fmalarda, del-zota olumlu bir g\u00fcvenlik profili sergiledi:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u00c7o\u011fu yan etki hafif ila orta \u015fiddetteydi.<\/strong><\/li>\n\n\n\n<li>Beklenmedik herhangi bir g\u00fcvenlik sinyali bildirilmedi.<\/li>\n\n\n\n<li><strong>Kreatin kinaz seviyelerinde \u00f6nemli d\u00fc\u015f\u00fc\u015fler g\u00f6zlemlendi.<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu bulgular, SAFARI44&#039;nin 3. faz klinik denemesinde devam eden ara\u015ft\u0131rmalar\u0131 desteklemektedir.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-the-safari-44-trial-matters\" class=\"wp-block-heading\">SAFARI44 Klinik Ara\u015ft\u0131rmas\u0131n\u0131n \u00d6nemi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Duchenne kas distrofisi i\u00e7in y\u00fcr\u00fct\u00fclen del-zota faz 3 SAFARI44 klinik \u00e7al\u0131\u015fmas\u0131, ekzon 44 atlamaya uygun hastalar i\u00e7in yeni bir hassas tedaviye y\u00f6nelik \u00f6nemli kan\u0131tlar sa\u011flayabilir.<\/strong> Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/upcoming-exon-44-skipping-therapies-for-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Tedavisinde Yakla\u015fan Ekzon 44 Atlama Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ba\u015far\u0131l\u0131 olmas\u0131 durumunda, del-zota \u015funlar\u0131 yapabilir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Distrofin \u00fcretimini art\u0131rmak<\/li>\n\n\n\n<li>Kas fonksiyonunu iyile\u015ftirmek<\/li>\n\n\n\n<li>Hastal\u0131\u011f\u0131n ilerlemesini geciktirmek<\/li>\n\n\n\n<li>DMD hastalar\u0131 i\u00e7in tedavi se\u00e7eneklerini geni\u015fletmek<\/li>\n<\/ul>\n\n\n\n<h2 id=\"in-which-countries-will-the-del-zota-phase-3-safari-44-clinical-trials-begin\" class=\"wp-block-heading\">Del-Zota Faz 3 SAFARI44 Klinik \u00c7al\u0131\u015fmalar\u0131 hangi \u00fclkelerde ba\u015flayacak?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Del-Zota'n\u0131n Faz 3 SAFARI44 klinik denemesi, k\u00fcresel bir klinik \u00e7al\u0131\u015fma olarak tasarlanm\u0131\u015ft\u0131r; bu da kat\u0131l\u0131mc\u0131 al\u0131m\u0131n\u0131n tek bir \u00fclke yerine birden fazla b\u00f6lgede ger\u00e7ekle\u015fece\u011fi anlam\u0131na gelir. \u00c7al\u0131\u015fman\u0131n Avrupa, Asya ve di\u011fer b\u00f6lgelerdeki \u00e7e\u015fitli \u00fclkelerde kat\u0131l\u0131mc\u0131 kaydetmesi beklenmektedir. Avidity Biosciences'nin, SAFARI44'\u00fcn ger\u00e7ekle\u015ftirilece\u011fi \u00fclkeleri duyurmas\u0131 beklenmektedir.<\/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>The del-zota phase 3 SAFARI44 clinical trial for Duchenne muscular dystrophy represents a major step toward developing targeted treatments for patients with exon-44\u2013amenable genetic mutations in Duchenne Muscular Dystrophy. Learn More: Mutations and Deletions Amenable to Exon 44 Skipping Therapies Presented at the Muscular Dystrophy Association Clinical &amp; Scientific Conference, the SAFARI44 study evaluates the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6751,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[126,137,298,141,127,246,77,178],"class_list":["post-6747","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-avidity-biosciences","tag-clinical-trial","tag-del-zota","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-exon-skipping","tag-exon-skipping-therapies"],"subtitle":"Duchenne kas distrofisi ara\u015ft\u0131rmalar\u0131nda yeni bir d\u00f6n\u00fcm noktas\u0131, Duchenne kas distrofisi i\u00e7in y\u00fcr\u00fct\u00fclen del-zota faz 3 SAFARI44 klinik \u00e7al\u0131\u015fmas\u0131yla ortaya \u00e7\u0131k\u0131yor. Bu \u00e7al\u0131\u015fma, bu nadir genetik hastal\u0131\u011fa sahip hastalarda distrofin \u00fcretimini geri kazand\u0131rmak ve kas fonksiyonunu iyile\u015ftirmek i\u00e7in tasarlanm\u0131\u015f, hedefli bir ekzon 44 atlama tedavisini i\u00e7eriyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6747","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=6747"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/6747\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/6751"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=6747"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=6747"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=6747"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}