{"id":3223,"date":"2025-03-17T10:57:18","date_gmt":"2025-03-17T07:57:18","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3223"},"modified":"2025-03-17T11:00:40","modified_gmt":"2025-03-17T08:00:40","slug":"roche-to-share-ongoing-work-on-elevidys-gene-therapy-at-muscular-dystrophy-association-mda-2025-conference","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/roche-to-share-ongoing-work-on-elevidys-gene-therapy-at-muscular-dystrophy-association-mda-2025-conference\/","title":{"rendered":"Roche, Kas Distrofisi Derne\u011fi (MDA) 2025 Konferans\u0131nda Elevidys Gen Terapisi \u00dczerindeki Devam Eden \u00c7al\u0131\u015fmalar\u0131n\u0131 Payla\u015facak"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Roche, Musk\u00fcler Distrofi Derne\u011fi&#039;nin (MDA) 2025 Konferans\u0131&#039;nda Elevidys Gen Terapisi \u00fczerindeki devam eden \u00e7al\u0131\u015fmalar\u0131n\u0131 tart\u0131\u015facak.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Son dakika Elevidys&#039; Embark iki y\u0131ll\u0131k verileri ve \u00c7al\u0131\u015fma 101, 102 ve Endeavor&#039;un birle\u015ftirilmi\u015f analizi, Duchenne kas distrofisi (DMD) olan erkek \u00e7ocuklarda motor fonksiyonun temel \u00f6l\u00e7\u00fcmlerinde klinik olarak anlaml\u0131 ve istatistiksel olarak anlaml\u0131 geli\u015fmeler oldu\u011funu g\u00f6stermi\u015ftir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Elevidys Devam Eden Klinik \u00c7al\u0131\u015fmalar<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys, EMBARK&#039;ta (A\u015fama III, NCT05096221 ) tedaviden iki y\u0131l sonra ve \u00c7al\u0131\u015fma 101&#039;in (A\u015fama I\/II, NCT03375164 , n=4), \u00c7al\u0131\u015fma 102&#039;nin (A\u015fama II, NCT03769116 , n=26) ve ENDEAVOR Kohort 1&#039;in (A\u015fama) Ib, NCT04626674 , n=20) birle\u015ftirilmi\u015f analizde tedaviden \u00fc\u00e7 y\u0131l sonra motor fonksiyonlar\u0131n\u0131n temel \u00f6l\u00e7\u00fcmlerinde anlaml\u0131 olarak anlaml\u0131 ve klinik olarak anlaml\u0131 iyile\u015ftirmeler g\u00f6sterildi, iyi e\u015fle\u015ftirilmi\u015f harici kontrol gruplar\u0131yla kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda. Toplu olarak, bu veriler Elevidys&#039;nin iletildi\u011fi, tedavi s\u0131ras\u0131nda d\u00f6rt ila sekiz ya\u015ftaki bireylerde ilerlemenin uzun vadeli stabilizasyonu veya yava\u015flamas\u0131 ile sonu\u00e7land\u0131\u011f\u0131 g\u00f6r\u00fclmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys, EMBARK&#039;ta (A\u015fama III, NCT05096221) tedaviden iki y\u0131l sonra ve \u00c7al\u0131\u015fma 101&#039;in (A\u015fama I\/II, NCT03375164, n=4), \u00c7al\u0131\u015fma 102&#039;nin (A\u015fama II, NCT03769116, n=26) ve ENDEAVOR Kohort 1&#039;in (A\u015fama Ib, NCT04626674, n=20) birle\u015ftirilmi\u015f analizinde tedaviden \u00fc\u00e7 y\u0131l sonra motor fonksiyonun temel \u00f6l\u00e7\u00fcmlerinde istatistiksel olarak anlaml\u0131 ve klinik olarak anlaml\u0131 iyile\u015fmeler g\u00f6sterdi, iyi e\u015fle\u015ftirilmi\u015f harici kontrol gruplar\u0131yla kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda. Toplu olarak, bu veriler Elevidys ile tedavinin, tedavi s\u0131ras\u0131nda d\u00f6rt ila sekiz ya\u015flar\u0131ndaki bireylerde hastal\u0131\u011f\u0131n ilerlemesinin uzun vadeli stabilizasyonu veya yava\u015flamas\u0131 ile sonu\u00e7land\u0131\u011f\u0131n\u0131 g\u00f6stermektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><a href=\"https:\/\/dmdwarrior.com\/tr\/roche-announces-new-results-from-the-embark-study-of-elevidys-in-outpatients-with-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"2612\" rel=\"noreferrer noopener\">EMBARK \u00e7al\u0131\u015fmas\u0131n\u0131n ikinci y\u0131l\u0131na ait \u00f6nemli veriler Ocak 2025&#039;te a\u00e7\u0131kland\u0131.<\/a> EMBARK \u00e7al\u0131\u015fmas\u0131nda iki y\u0131ll\u0131k s\u00fcre zarf\u0131nda yeni g\u00fcvenlik sinyallerine rastlanmad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys&#039;nin hastal\u0131k ilerlemesi \u00fczerindeki etkisini daha ileri de\u011ferlendirmek i\u00e7in, kas sa\u011fl\u0131\u011f\u0131 ve kas patolojisindeki de\u011fi\u015fiklikler EMBARK birinci b\u00f6l\u00fcmdeki bireylerin bir alt grubunda manyetik rezonans g\u00f6r\u00fcnt\u00fcleme (MRI) ile de\u011ferlendirildi. 52. haftada, sonu\u00e7lar Elevidys ile tedavi edilen hastalarda plasebo ile tedavi edilen hastalara k\u0131yasla hastal\u0131k ilerlemesinin stabilize oldu\u011funu veya yava\u015flad\u0131\u011f\u0131n\u0131 g\u00f6sterdi. 104. haftada, ba\u015flang\u0131\u00e7 seviyesinden itibaren MRI de\u011fi\u015fiklikleri, 52. haftada kaslar ve kas gruplar\u0131 aras\u0131nda plaseboya k\u0131yasla Elevidys&#039;yi genel olarak desteklemeye devam etti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Devam\u0131n\u0131 oku: <a href=\"https:\/\/dmdwarrior.com\/tr\/clinical-trials-review-of-elevidys-gene-therapy-is-elevidys-cost-worthy\/\" target=\"_blank\" data-type=\"post\" data-id=\"3027\" rel=\"noreferrer noopener\">Elevidys Gen Terapisi: Fiyat\u0131na De\u011fer mi?<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Elevidys i\u00e7in Pazarlama Yetkilendirmesi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys, ABD, Birle\u015fik Arap Emirlikleri (BAE), Katar, Kuveyt, Bahreyn ve Umman&#039;da ayakta tedavi durumlar\u0131na bak\u0131lmaks\u0131z\u0131n d\u00f6rt ya\u015f ve \u00fczeri Duchenne hastas\u0131 ki\u015filer i\u00e7in onaylanm\u0131\u015ft\u0131r. Elevidys ayr\u0131ca Brezilya ve \u0130srail&#039;de d\u00f6rt ila yedi ya\u015f aras\u0131 ayakta tedavi g\u00f6ren bireylerin tedavisi i\u00e7in onaylanm\u0131\u015ft\u0131r. Ayr\u0131ca Avrupa \u0130la\u00e7 Ajans\u0131&#039;na (EMA) ve Japonya, \u0130svi\u00e7re, Singapur, Hong Kong ve Suudi Arabistan&#039;daki d\u00fczenleyici makamlara ba\u015fvurular yap\u0131lm\u0131\u015ft\u0131r. Bug\u00fcne kadar 600&#039;den fazla hasta Elevidys ile tedavi edilmi\u015ftir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/why-isnt-roche-applying-marketing-authorization-in-every-country-for-elevidys-gene-therapy\/\">Roche Neden Her \u00dclkede Elevidys Gen Terapisi \u0130\u00e7in Pazarlama Yetkisi Ba\u015fvurusunda Bulunmuyor?<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>At the Muscular Dystrophy Association (MDA) 2025 Conference, Roche will discuss their ongoing work on Elevidys Gene Therapy. Late-breaking oral on Elevidys\u2019 Embark two-year data and pooled analysis of Study 101, 102 and Endeavor, demonstrated clinically meaningful and statistically significant improvements across key measures of motor function in boys with Duchenne muscular dystrophy (DMD) Elevidys [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3224,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[33,61,297,203],"class_list":["post-3223","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevidys","tag-gene-therapy","tag-muscular-dystrophy-association","tag-roche"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3223","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=3223"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3223\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3224"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3223"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3223"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3223"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}