{"id":3477,"date":"2025-04-01T17:31:55","date_gmt":"2025-04-01T14:31:55","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3477"},"modified":"2025-04-04T10:01:49","modified_gmt":"2025-04-04T07:01:49","slug":"three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe\/","title":{"rendered":"Avrupa&#039;da Elevidys Gen Terapisinin Devam Eden \u00dc\u00e7 Klinik \u00c7al\u0131\u015fmas\u0131 Ge\u00e7ici Olarak Durduruldu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Sarepta taraf\u0131ndan geli\u015ftirilen ve Roche taraf\u0131ndan pazarlanan bir gen terapisi olan ELEVIDYS&#039;in (delandistrogene moxeparvovec-rokl) devam eden \u00fc\u00e7 klinik denemesi, Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) taraf\u0131ndan ge\u00e7ici olarak durduruldu. Beklenen ancak istenmeyen bu haber, Duchenne kas distrofisi (DMD) toplulu\u011funda derin bir \u00fcz\u00fcnt\u00fcye neden oldu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Duraklat\u0131lan \u00e7al\u0131\u015fmalar aras\u0131nda, 8 ila 17 ya\u015f aras\u0131ndaki erkek \u00e7ocuklar\u0131nda Elevidys&#039;yi de\u011ferlendiren Sarepta destekli faz 3 Envision \u00e7al\u0131\u015fmas\u0131; bebeklerde ve yenido\u011fanlarda Elevidys&#039;yi ara\u015ft\u0131ran Roche&#039;un faz 2 Envol \u00e7al\u0131\u015fmas\u0131; ve Sarepta&#039;n\u0131n \u00c7al\u0131\u015fma 104&#039;\u00fc, belirli bir serotipe kar\u015f\u0131 \u00f6nceden var olan antikorlar\u0131 olan 4 ila 9 ya\u015f aras\u0131ndaki erkek \u00e7ocuklar\u0131nda Elevidys&#039;ye y\u00f6nelik faz 1 ara\u015ft\u0131rmas\u0131 yer al\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Roche, Sarepta ve EMA&#039;den \u00f6nce bu duyuruyu yay\u0131nlayan ilk topluluk T\u00fcrk orta\u011f\u0131m\u0131zd\u0131 &gt;&gt;&gt; <a href=\"https:\/\/dmd.arti.net\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD Dayan\u0131\u015fma<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Roche, topluma bir mektup g\u00f6nderdi, mektubu burada okuyabilirsiniz.<a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/March-2025-Roche-DMD-Temporary-Clinical-Study-Halt-Community-Letter_31Mar25.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">PDF<\/a>)<\/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=\"#why-were-elevidys-clinical-trials-halted\">Elevidys Klinik Ara\u015ft\u0131rmalar\u0131 Neden Durduruldu?<\/a><ul><li><a href=\"#srp-9001-104\">SRP-9001-104 Ge\u00e7ici Olarak Durduruldu<\/a><\/li><li><a href=\"#envol-study-302\">ENVOL (\u00c7al\u0131\u015fma 302) Ge\u00e7ici Olarak Durduruldu<\/a><\/li><li><a href=\"#srp-9001-303\">SRP-9001-303 Ge\u00e7ici Olarak Durduruldu<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-were-elevidys-clinical-trials-halted\">Elevidys Klinik Ara\u015ft\u0131rmalar\u0131 Neden Durduruldu?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">18 Mart 2025&#039;te Elevidys inf\u00fczyonu alan Duchenne kas distrofisi hastas\u0131 16 ya\u015f\u0131ndaki bir erkek \u00e7ocu\u011funun \u00f6l\u00fcm\u00fcnden sonra, $3 milyon dolarl\u0131k ticari fiyatla pazarlanan gen terapisinin g\u00f6r\u00fcn\u00fcm\u00fc aniden de\u011fi\u015fti.<a href=\"https:\/\/dmdwarrior.com\/tr\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\">Devam\u0131n\u0131 oku<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys ald\u0131ktan sonra akut karaci\u011fer yetmezli\u011fi (ALF) geli\u015ftiren bir ABD gencinin kesin \u00f6l\u00fcm nedeni belirlenene kadar, Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) \u00e7al\u0131\u015fmalar\u0131n ask\u0131ya al\u0131nmas\u0131n\u0131 talep etti. Hasta ge\u00e7en ay vefat etti\u011finde, Sarepta Elevidys&#039;nin etiketini g\u00fcvenlik sinyalini i\u00e7erecek \u015fekilde de\u011fi\u015ftirece\u011fini duyurdu.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-104\">SRP-9001-104 Ge\u00e7ici Olarak Durduruldu<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"113\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg\" alt=\"SRP-9001-104 Elevidys Gen Terapisinin Klinik Denemesi Avrupa&#039;da Ge\u00e7ici Olarak Durduruldu\" class=\"wp-image-3480\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-300x44.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-18x3.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-150x22.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-696x103.jpg 696w\" sizes=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Kaynak: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512624-11-00?lang=en\" target=\"_blank\" rel=\"noopener\">SRP-9001-104&#039;\u00fcn Klinik Denemesi<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"envol-study-302\">ENVOL (\u00c7al\u0131\u015fma 302) Ge\u00e7ici Olarak Durduruldu<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"272\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg\" alt=\"ENVOL (\u00c7al\u0131\u015fma 302) Elevidys Gen Terapisinin Klinik Denemesi Avrupa&#039;da Ge\u00e7ici Olarak Durduruldu\" class=\"wp-image-3481\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-300x107.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-18x6.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-150x54.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-696x248.jpg 696w\" sizes=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Kaynak: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2023-509901-57-00?lang=en\" target=\"_blank\" rel=\"noopener\">SRP-9001-302&#039;nin Klinik Denemesi<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-303\">SRP-9001-303 Ge\u00e7ici Olarak Durduruldu<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"768\" height=\"313\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg\" alt=\"SRP-9001-303 Elevidys Gen Terapisinin Klinik Denemesi Avrupa&#039;da Ge\u00e7ici Olarak Durduruldu\" class=\"wp-image-3482\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-300x122.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-18x7.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-150x61.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-696x284.jpg 696w\" sizes=\"auto, (max-width: 768px) 100vw, 768px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Kaynak: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512626-28-00?lang=en\" target=\"_blank\" rel=\"noopener\">SRP-9001-303&#039;\u00fcn Klinik Denemesi<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-warriors-turkish-representative-shares-his-views-on-elevidys-gene-therapy-is-it-effective-and-why-is-it-expensive\/\">DMD WarrioR&#039;un T\u00fcrkiye Temsilcisi Elevidys Gen Terapisi Hakk\u0131ndaki G\u00f6r\u00fc\u015flerini Payla\u015f\u0131yor.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the European Medicines Agency (EMA). This expected but not unwelcome news has caused deep sadness in the Duchenne muscular dystrophy (DMD) community. The paused trials include the Sarepta-sponsored phase 3 Envision study, [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3478,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,33,179,91,84,213,61,203,65],"class_list":["post-3477","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-elevidys","tag-elevidys-gene-therapy","tag-ema","tag-europe","tag-european-medicines-agency","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3477","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=3477"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3477\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3478"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3477"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3477"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3477"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}