{"id":3230,"date":"2025-03-17T14:28:46","date_gmt":"2025-03-17T11:28:46","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3230"},"modified":"2025-03-17T14:28:48","modified_gmt":"2025-03-17T11:28:48","slug":"avidity-biosciences-announces-positive-topline-del-zota-data-showing-consistent-statistically-significant-improvements-in-dystrophin","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/avidity-biosciences-announces-positive-topline-del-zota-data-showing-consistent-statistically-significant-improvements-in-dystrophin\/","title":{"rendered":"Avidity Biosciences, Distrofin&#039;de Tutarl\u0131, \u0130statistiksel Olarak Anlaml\u0131 \u0130yile\u015fmeler G\u00f6steren Pozitif Topline Del-zota Verilerini Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences, Ekson 44 Atlamaya Uygun Duchenne Musk\u00fcler Distrofisi Olan Faz 1\/2 EXPLORE44\u00ae Deneme Kat\u0131l\u0131mc\u0131lar\u0131nda Distrofin, Ekson Atlama ve Kreatin Kinazda Tutarl\u0131, \u0130statistiksel Olarak Anlaml\u0131 \u0130yile\u015fmeler G\u00f6steren Olumlu Topline Del-zota Verilerini Duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Veriler, 16-19 Mart 2025 tarihleri aras\u0131nda Dallas, Teksas&#039;ta d\u00fczenlenecek olan 2025 Musk\u00fcler Distrofi Derne\u011fi (MDA) Klinik ve Bilimsel Konferans\u0131&#039;nda s\u00f6zl\u00fc ve poster sunumuyla vurgulanacakt\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Avidity Biosciences EXPLORE44 (Del-zota) Klinik Denemeleri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cDel-zota, distrofin \u00fcretiminde \u00f6nemli bir art\u0131\u015f ve sadece \u00fc\u00e7 dozdan sonra serum kreatin kinaz seviyelerinde \u00f6nemli bir azalma ve neredeyse normale yak\u0131n bir d\u00fc\u015f\u00fc\u015f dahil olmak \u00fczere birden fazla \u00f6l\u00e7\u00fcmde dikkate de\u011fer geli\u015fmeler g\u00f6sterdi. Bu sonu\u00e7lar\u0131n bu kadar k\u0131sa bir zaman diliminde tutarl\u0131 olmas\u0131, uygun g\u00fcvenlik ve tolere edilebilirlikle birle\u015fti\u011finde, del-zota&#039;nin ekzon 44 atlamaya uygun genetik varyantlara sahip DMD&#039;li bireyler i\u00e7in \u00e7\u0131\u011f\u0131r a\u00e7an bir tedavi olma potansiyelini vurguluyor,\u201d diyor Arkansas T\u0131p Bilimleri \u00dcniversitesi ve Arkansas \u00c7ocuk Hastanesi Pediatri Do\u00e7enti Dr. Aravindhan Veerapandiyan.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cBu sonu\u00e7lar, kas b\u00fct\u00fcnl\u00fc\u011f\u00fcn\u00fc koruyabilecek ve muhtemelen bu hastal\u0131kla ili\u015fkili kas zay\u0131fl\u0131\u011f\u0131 ve fonksiyon kayb\u0131n\u0131n ilerlemesini \u00f6nleyebilecek veya geciktirebilecek hedefli tedavilere acil ihtiya\u00e7 duyan DMD&#039;den etkilenen hastalar ve aileleri i\u00e7in yeni bir umut getiriyor.\u201d<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Del-Zota nedir?<\/h2>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"576\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1024x576.jpg\" alt=\"Avidity Biosciences Del-zota EXPLORE44 ekzon 44 atlama\" class=\"wp-image-3234\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1024x576.jpg 1024w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-300x169.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-768x432.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1536x864.jpg 1536w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-18x10.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-747x420.jpg 747w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-150x84.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-696x392.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota-1068x601.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/Avidity-Biosciences-Del-Zota.jpg 1920w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Del-zota, distrofin geninin 44. eksonunu \u00f6zel olarak atlamak ve neredeyse tam uzunlukta distrofin \u00fcretimini sa\u011flamak i\u00e7in iskelet ve kalp kas\u0131 dokusuna fosforodiamidat morfolino oligomerleri (PMO&#039;lar) iletmek \u00fczere tasarlanm\u0131\u015ft\u0131r. Del-zota, ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) ve Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) taraf\u0131ndan Yetim stat\u00fcs\u00fcne lay\u0131k g\u00f6r\u00fclm\u00fc\u015ft\u00fcr. FDA ayr\u0131ca del-zota Nadir Pediatrik Hastal\u0131k ve H\u0131zl\u0131 Yol stat\u00fclerine de lay\u0131k g\u00f6r\u00fclm\u00fc\u015ft\u00fcr. Del-zota, Avidity&#039;de \u015fu anda DMD tedavisi i\u00e7in geli\u015ftirilmekte olan birden fazla AOC&#039;nin ilkidir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>MDA&#039;da sunulan veriler, del-zota&#039;nin hem 5 mg\/kg hem de 10 mg\/kg kohortlar\u0131ndaki t\u00fcm parametrelerde tutarl\u0131 verileri vurgulamaktad\u0131r:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0130skelet kas\u0131nda yakla\u015f\u0131k 200nM&#039;lik doku konsantrasyonlar\u0131yla sonu\u00e7lanan PMO&#039;lar\u0131n hedeflenen iletimi;<\/li>\n\n\n\n<li>Ekson 44 atlamada yakla\u015f\u0131k 40%&#039;de istatistiksel olarak anlaml\u0131 art\u0131\u015flar;<\/li>\n\n\n\n<li>Distrofin \u00fcretiminde normalin yakla\u015f\u0131k 25%&#039;si kadar istatistiksel olarak anlaml\u0131 art\u0131\u015f ve normalin 58%&#039;sine kadar toplam distrofin geri kazan\u0131ld\u0131;<\/li>\n\n\n\n<li>Kreatin kinaz d\u00fczeylerinde ba\u015flang\u0131\u00e7 seviyesine k\u0131yasla 80%&#039;den daha b\u00fcy\u00fck azalmalarla normale yak\u0131n azalma:\n<ul class=\"wp-block-list\">\n<li>Benzer \u015fekilde, plasebo kat\u0131l\u0131mc\u0131lar\u0131nda del-zota tedavisi sonras\u0131nda kreatin kinaz d\u00fczeylerinde neredeyse normale yak\u0131n bir d\u00fc\u015f\u00fc\u015f g\u00f6r\u00fcld\u00fc;<\/li>\n\n\n\n<li>EXPLORE44-OLE denemesinde, bir y\u0131la kadar devam eden tedaviyle kreatin kinaz d\u00fczeylerinde \u00f6nemli azalmalar s\u00fcrd\u00fcr\u00fcld\u00fc; ve<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Del-zota her iki dozda da olumlu g\u00fcvenlik ve tolere edilebilirlik g\u00f6sterdi; tedaviyle ortaya \u00e7\u0131kan yan etkilerin \u00e7o\u011fu hafif veya orta \u015fiddetteydi.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Musk\u00fcler Distrofi Tedavisi \u0130\u00e7in Geli\u015ftirilen Yeni Nesil Ekzon Atlama Terapileri<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Avidity Biosciences Announces Positive Topline Del-zota Data Showing Consistent, Statistically Significant Improvements in Dystrophin, Exon Skipping, and Creatine Kinase in Phase 1\/2 EXPLORE44\u00ae Trial Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping. The data will be highlighted in an oral and poster presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3232,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,141,127,246,299],"class_list":["post-3230","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","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\/3230","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=3230"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3230\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3232"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3230"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3230"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3230"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}