{"id":3720,"date":"2025-04-18T14:28:26","date_gmt":"2025-04-18T11:28:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3720"},"modified":"2025-04-18T14:39:11","modified_gmt":"2025-04-18T11:39:11","slug":"del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping\/","title":{"rendered":"Del-zota (EXPLORE44), Ekzon 44 Atlamaya Uygun Hastalarda Neredeyse Y\u00fczde 30 Normal Distrofin \u00dcretimi Sa\u011fl\u0131yor"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Distrofin okuma \u00e7er\u00e7evesini onarmak ve i\u015flevsel ve dahili olarak kesilmi\u015f bir distrofin proteini \u00fcretmek amac\u0131yla geli\u015ftirilen Del-zota&#039;y\u0131 de\u011ferlendirmek i\u00e7in \u015fu anda bir faz 1\/2 EXPLORE44 \u00e7al\u0131\u015fmas\u0131 (NCT05670730) y\u00fcr\u00fct\u00fclmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">16-19 Mart tarihleri aras\u0131nda Dallas, Teksas&#039;ta ger\u00e7ekle\u015fen 2025 Muscular Dystrophy Association (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda, randomize, \u00e7ift k\u00f6r ve plasebo ile kontrol edilen bu denemenin sonu\u00e7lar\u0131 yak\u0131n zamanda sunuldu. Bulgulara dayanarak, Del-zota&#039;n\u0131n distrofin \u00fcretimini ve ekzon atlamay\u0131 art\u0131rma ve ayn\u0131 anda kreatin kinaz (CK) say\u0131mlar\u0131n\u0131 d\u00fc\u015f\u00fcrme yetene\u011fine sahip oldu\u011fu g\u00f6sterildi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity&#039;deki ba\u015f bilim g\u00f6revlisi Dr. Michael Flanagan, Del-zota ve faz 1\/2 sonu\u00e7lar\u0131n\u0131 daha ayr\u0131nt\u0131l\u0131 olarak g\u00f6r\u00fc\u015fmek \u00fczere NeurologyLive taraf\u0131ndan r\u00f6portaj yap\u0131ld\u0131. Ekson 44&#039;\u00fc hedef alan benzersiz da\u011f\u0131t\u0131m y\u00f6ntemini ve halihaz\u0131rda onaylanm\u0131\u015f ila\u00e7lardan ve ara\u015ft\u0131rma alt\u0131ndaki di\u011fer ila\u00e7lardan nas\u0131l farkl\u0131 oldu\u011funu a\u00e7\u0131klad\u0131. Flanagan&#039;\u0131n ayr\u0131ca \u00f6nizlemesini verdi\u011fi ilac\u0131n gelecekteki planlar\u0131 aras\u0131nda do\u011frulay\u0131c\u0131 bir faz 3 denemesi, a\u00e7\u0131k etiketli bir uzatma ve olas\u0131 bir biyolojik lisans ba\u015fvurusu yer al\u0131yor.<\/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=\"#phase-1-2-explore-44-trial-design\">Faz 1\/2 EXPLORE 44 Denemesi<\/a><\/li><li><a href=\"#safety-and-tolerability\">G\u00fcvenlik ve Tolerans<\/a><\/li><li><a href=\"#biomarker-outcomes\">Biyobelirte\u00e7 Sonu\u00e7lar\u0131<\/a><\/li><li><a href=\"#creatine-kinase-ck-levels\">Kreatin Kinaz (CK) Seviyeleri<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"phase-1-2-explore-44-trial-design\">Faz 1\/2 EXPLORE 44 Denemesi<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Randomize, plasebo kontroll\u00fc, \u00e7ift k\u00f6r deneme<\/li>\n\n\n\n<li>DMD44&#039;l\u00fc 24 kat\u0131l\u0131mc\u0131 (ya\u015flar\u0131 7-27, hem y\u00fcr\u00fcyebilen hem de y\u00fcr\u00fcyemeyen)<\/li>\n\n\n\n<li>Tedavi kollar\u0131: 6 haftada bir 5 mg\/kg, 8 haftada bir 10 mg\/kg ve plasebo (3:1 randomizasyon)<\/li>\n\n\n\n<li>Ba\u015flang\u0131\u00e7ta ve \u00fc\u00e7\u00fcnc\u00fc dozdan bir ay sonra ger\u00e7ekle\u015ftirilen kas biyopsileri<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"safety-and-tolerability\">G\u00fcvenlik ve Tolerans<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c7o\u011fu olumsuz olay\u0131n hafif ila orta \u015fiddette oldu\u011fu olumlu g\u00fcvenlik profili<\/li>\n\n\n\n<li>En s\u0131k g\u00f6r\u00fclen yan etkiler: i\u015flem a\u011fr\u0131s\u0131 ve ba\u015f a\u011fr\u0131s\u0131<\/li>\n\n\n\n<li>\u0130ki kat\u0131l\u0131mc\u0131 \u00e7al\u0131\u015fmay\u0131 b\u0131rakt\u0131: 1&#039;i anafilaksi nedeniyle, 1&#039;i orta d\u00fczeyde inf\u00fczyonla ili\u015fkili reaksiyon nedeniyle<\/li>\n\n\n\n<li>Semptomatik hemoglobin de\u011fi\u015fikli\u011fi yok, hipomagnezemi yok, \u00f6l\u00fcm yok<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"biomarker-outcomes\">Biyobelirte\u00e7 Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Her iki dozaj grubunda da tutarl\u0131 ve y\u00fcksek PMO kas konsantrasyonu<\/li>\n\n\n\n<li>\u00d6nemli ekzon 44 atlamas\u0131: 5 mg\/kg grubunda 37%, 10 mg\/kg grubunda 43%<\/li>\n\n\n\n<li>Distrofin \u00fcretimi ortalama olarak normal seviyelerin yakla\u015f\u0131k 25%&#039;sine y\u00fckseldi<\/li>\n\n\n\n<li>T\u00fcm ekzon 44 uyumlu genotiplerde g\u00f6zlemlenen 58%&#039;ye kadar normal distrofin seviyeleri<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"creatine-kinase-ck-levels\">Kreatin Kinaz (CK) Seviyeleri<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Tedavi edilen kat\u0131l\u0131mc\u0131larda CK d\u00fczeyleri s\u00fcrekli olarak normale yak\u0131n bir seviyeye d\u00fc\u015ft\u00fc<\/li>\n\n\n\n<li>CK d\u00fc\u015f\u00fc\u015fleri 5 mg\/kg grubunda 70-140. g\u00fcn ve 10 mg\/kg grubunda 84-140. g\u00fcn aras\u0131nda normalin \u00fcst s\u0131n\u0131r\u0131na yak\u0131n bir seviyede s\u00fcrd\u00fcr\u00fcld\u00fc<\/li>\n\n\n\n<li>Ba\u015flang\u0131\u00e7 seviyesine k\u0131yasla CK d\u00fczeylerinde 80%&#039;den daha b\u00fcy\u00fck azalma<\/li>\n\n\n\n<li>A\u00e7\u0131k etiketli uzatmada 1 y\u0131la kadar s\u00fcren indirimler<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">Yeni Nesil Ekzon Atlama Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>A phase 1\/2 EXPLORE44 study (NCT05670730) is now being conducted to evaluate Del-zota, which was developed with the intention of restoring the dystrophin reading frame and producing a dystrophin protein that is functional and internally truncated. At the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, which took place in Dallas, Texas, from March [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3722,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[126,298,141,127,246,299],"class_list":["post-3720","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-avidity-biosciences","tag-del-zota","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3720","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=3720"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3720\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3722"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3720"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3720"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3720"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}