{"id":5095,"date":"2025-11-04T10:21:05","date_gmt":"2025-11-04T07:21:05","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5095"},"modified":"2025-11-04T10:21:20","modified_gmt":"2025-11-04T07:21:20","slug":"agamree-vamorolone-five-year-data","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/agamree-vamorolone-five-year-data\/","title":{"rendered":"DMD&#039;li Hastalarda AGAMREE&#039;nin (vamorolon) Be\u015f Y\u0131ll\u0131k Verileri, Standart Bak\u0131m Kortikosteroidleriyle Kar\u015f\u0131la\u015ft\u0131r\u0131labilir Etkinlikle Geli\u015fmi\u015f G\u00fcvenlik Profili G\u00f6stermektedir"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Santhera, Duchenne kas distrofisi (DMD) hastalar\u0131nda AGAMREE&#039;yi (vamorolon) de\u011ferlendiren devam eden, a\u00e7\u0131k etiketli, \u00e7ok merkezli GUARDIAN \u00e7al\u0131\u015fmas\u0131ndan al\u0131nan ilk de\u011ferlendirmeler de dahil olmak \u00fczere uzun vadeli verilerin analizinden olumlu ilk sonu\u00e7lar bildirdi. &gt;&gt;&gt; <a href=\"https:\/\/dmdwarrior.com\/tr\/what-is-agamree-vamorolone-what-is-agamree-used-for\/\" target=\"_blank\" data-type=\"post\" data-id=\"2333\" rel=\"noreferrer noopener\">Agamree nedir?<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u00d6ne \u00c7\u0131kan Sonu\u00e7lar<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>5 y\u0131l boyunca standart bak\u0131m kortikosteroidlerine k\u0131yasla kar\u015f\u0131la\u015ft\u0131r\u0131labilir uzun vadeli etkinlik<\/li>\n\n\n\n<li>Standart bak\u0131m kortikosteroidlerinde s\u0131kl\u0131kla g\u00f6r\u00fclen b\u00fcy\u00fcme bask\u0131lanmas\u0131n\u0131n aksine, normal b\u00fcy\u00fcme korunmu\u015ftur<\/li>\n\n\n\n<li>AGAMREE ile bildirilen vertebral k\u0131r\u0131k oran\u0131 \u00f6nemli \u00f6l\u00e7\u00fcde daha d\u00fc\u015f\u00fckt\u00fcr<\/li>\n\n\n\n<li>Kortikosteroid kullanan DMD hastalar\u0131nda normalde g\u00f6r\u00fclenden daha d\u00fc\u015f\u00fck katarakt g\u00f6r\u00fclme s\u0131kl\u0131\u011f\u0131<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">110 hasta analiz edildi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Uzun vadeli analiz, klinik \u00e7al\u0131\u015fmalarda d\u00f6rt ila yedi ya\u015flar\u0131 aras\u0131nda AGAMREE tedavisine ba\u015flayan ve GUARDIAN \u00e7al\u0131\u015fmas\u0131 da dahil olmak \u00fczere \u00e7e\u015fitli eri\u015fim programlar\u0131yla tedaviye devam eden kortikosteroidsiz hastalar\u0131 i\u00e7eriyordu. <strong>110 hastaya kadar veri analiz edildi ve hasta say\u0131lar\u0131 veri bulunabilirli\u011fine ba\u011fl\u0131 olarak analize g\u00f6re de\u011fi\u015fti. Bu uzun vadeli analizde, hastalar sekiz y\u0131la kadar AGAMREE alm\u0131\u015f ve ortalama takip s\u00fcresi yakla\u015f\u0131k be\u015f y\u0131l olmu\u015ftur.<\/strong> G\u00f6zlem s\u00fcresi boyunca hastalar\u0131n \u00e7o\u011fu ger\u00e7ek d\u00fcnya klinik ortamlar\u0131nda daha y\u00fcksek dozlarda (4-6 mg\/kg\/g\u00fcn) kald\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>AGAMREE ile tedavi edilen hastalar, uzun s\u00fcreli takipte motor fonksiyonlar\u0131n\u0131 korudular ve standart bak\u0131m kortikosteroidlerine k\u0131yasla y\u00fcr\u00fcme kayb\u0131 s\u00fcresiyle \u00f6l\u00e7\u00fclen kal\u0131c\u0131 etkililik g\u00f6sterdiler (p=0,91)<\/strong>. \u00d6nceden belirlenmi\u015f alt grup analizlerinde g\u00fcnl\u00fck deflazakort veya prednizon kullan\u0131m\u0131na g\u00f6re herhangi bir fark g\u00f6zlenmedi.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">G\u00fcvenlik ve tolere edilebilirlik<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00d6nemlisi, veriler geleneksel kortikosteroidlere k\u0131yasla farkl\u0131 bir g\u00fcvenlik ve tolere edilebilirlik profilini desteklemeye devam ediyor. <strong>AGAMREE ile tedavi edilen hastalarda vertebral k\u0131r\u0131k oran\u0131 \u00f6nemli \u00f6l\u00e7\u00fcde daha d\u00fc\u015f\u00fckt\u00fc (p=0,0061), standart bak\u0131m kortikosteroidlerinde g\u00f6r\u00fclen bodurluk olmadan normal b\u00fcy\u00fcmeyi s\u00fcrd\u00fcrd\u00fcler (p&lt;0,0001) ve glukokortikoidlerle tedavi edilenlere g\u00f6re daha az katarakt vakas\u0131 g\u00f6r\u00fcld\u00fc; deflazakort ile kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda belirgin \u015fekilde daha d\u00fc\u015f\u00fck bir insidans g\u00f6r\u00fcld\u00fc (p&lt;0,015).<\/strong> Ayr\u0131ca, bug\u00fcne kadar herhangi bir glokom vakas\u0131 g\u00f6zlemlenmemi\u015ftir. Ortalama olarak, boyla e\u015fle\u015ftirildi\u011finde BMI veya kiloda de\u011fi\u015fiklik g\u00f6r\u00fclmemi\u015ftir ve yeni bir g\u00fcvenlik sinyali g\u00f6zlemlenmemi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ayr\u0131nt\u0131l\u0131 sonu\u00e7lar, 2026&#039;n\u0131n ilk \u00e7eyre\u011finde d\u00fczenlenecek b\u00fcy\u00fck bir uluslararas\u0131 bilimsel konferansta sunulmak \u00fczere sunulmaktad\u0131r. Standart bilimsel uygulamalara uygun olarak, konferans sunumunun ard\u0131ndan, organizat\u00f6rler inceleme ve sunum s\u00fcre\u00e7lerini tamamlad\u0131ktan sonra, t\u00fcm veriler kamuoyuyla payla\u015f\u0131lacakt\u0131r. Ayr\u0131ca, \u00f6n\u00fcm\u00fczdeki \u00fc\u00e7 y\u0131l i\u00e7inde GUARDIAN \u00e7al\u0131\u015fmas\u0131ndan elde edilen daha kapsaml\u0131 veri setlerinin, \u00fcst ekstremite fonksiyonu, ergenlik geli\u015fimi, g\u00f6z sa\u011fl\u0131\u011f\u0131 ve kalp fonksiyonu ile di\u011fer ilgili parametrelerin de\u011ferlendirilmesi de dahil olmak \u00fczere daha geni\u015f bir etkinlik ve g\u00fcvenlik sonu\u00e7lar\u0131 k\u00fcmesine odaklanmas\u0131 planlanmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bu Sayfay\u0131 Takip Edin<\/strong> >>> <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>Santhera reported positive topline results from an analysis of long-term data, including first assessments from the ongoing, open-label, multicenter GUARDIAN study evaluating AGAMREE (vamorolone) in patients with Duchenne muscular dystrophy (DMD). >>> What is Agamree? Featured Results 110 patients were analyzed The long-term analysis included corticosteroid-na\u00efve patients who began AGAMREE treatment between four and seven [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5097,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[42,124,41,43],"class_list":["post-5095","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-agamree","tag-corticosteroid","tag-santhera","tag-vamorolone"],"subtitle":"Agamree'nin (vamorolon) 5 y\u0131ll\u0131k verileri, geleneksel kortikosteroidlere k\u0131yasla farkl\u0131 bir g\u00fcvenlik ve tolere edilebilirlik profilini desteklemeye devam ediyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5095","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=5095"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5095\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5097"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5095"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5095"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5095"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}