{"id":3925,"date":"2025-06-15T12:00:44","date_gmt":"2025-06-15T09:00:44","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3925"},"modified":"2025-06-15T12:00:45","modified_gmt":"2025-06-15T09:00:45","slug":"sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients\/","title":{"rendered":"Sarepta, Duchenne Musk\u00fcler Distrofisi i\u00e7in Elevidys Gen Terapisinin y\u00fcr\u00fcyemeyen hastalarda kullan\u0131lmayaca\u011f\u0131n\u0131 bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Duchenne kas distrofisi hastalar\u0131 i\u00e7in onaylanm\u0131\u015f tek gen terapisi olan ELEVIDYS (delandistrogene moxeparvovec-rokl), nadir hastal\u0131klar i\u00e7in hassas genetik t\u0131pta lider olan Sarepta Therapeutics&#039;den bir g\u00fcvenlik g\u00fcncellemesi ald\u0131. \u015eirket ayr\u0131ca, hareket edemeyen hastalarda g\u00fcvenlik profilini iyile\u015ftirmek i\u00e7in ald\u0131\u011f\u0131 \u00f6nlemleri de g\u00f6r\u00fc\u015ft\u00fc. \u00d6l\u00fcmle sonu\u00e7lanan ikinci bir belgelenmi\u015f akut karaci\u011fer yetmezli\u011fi (ALF) vakas\u0131n\u0131n ard\u0131ndan bu \u00f6nlemler al\u0131nd\u0131. \u015eimdiye kadar, her iki ALF vakas\u0131 da hareket edemeyen Duchenne hastalar\u0131yd\u0131. Sarepta, etkilenen ailelere ve bak\u0131c\u0131lara en i\u00e7ten taziyelerini iletiyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Roche ayn\u0131 zamanda bug\u00fcn, hem klinik hem de ticari ba\u011flamda, ya\u015fa bak\u0131lmaks\u0131z\u0131n, y\u00fcr\u00fcyemeyen Duchenne kas distrofisi (DMD) hastalar\u0131nda derhal y\u00fcr\u00fcrl\u00fc\u011fe girecek olan ELEVIDYS (delandistrojen moxeparvovek) i\u00e7in revize edilmi\u015f doz y\u00f6nergelerini a\u00e7\u0131klad\u0131. <strong>Elevidys art\u0131k ticari ortamda y\u00fcr\u00fcyemeyen hastalara uygulanmamal\u0131d\u0131r.<\/strong> Klinik deneme ortam\u0131ndaki hareketsiz hastalar\u0131n kayd\u0131 ve dozlamas\u0131, ara\u015ft\u0131rma protokol\u00fc ek risk azaltma stratejileri (ba\u011f\u0131\u015f\u0131kl\u0131k d\u00fczenleyici ila\u00e7lar gibi) i\u00e7ermedi\u011fi s\u00fcrece derhal durdurulacakt\u0131r. Bilgiler, hasta bak\u0131m\u0131n\u0131n derhal de\u011fi\u015ftirilebilmesi i\u00e7in doktorlar, ara\u015ft\u0131rmac\u0131lar ve sa\u011fl\u0131k otoriteleriyle payla\u015f\u0131l\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Sarepta, gen tedavisinin ard\u0131ndan ikinci karaci\u011fer yetmezli\u011fi \u00f6l\u00fcm vakas\u0131n\u0131 bildirdi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Bu karar, y\u00fcr\u00fcyemeyen hastalarda iki \u00f6l\u00fcmc\u00fcl akut karaci\u011fer yetmezli\u011fi (ALF) vakas\u0131n\u0131n dikkatlice de\u011ferlendirilmesinin ard\u0131ndan geldi. Elevidys ve di\u011fer AAV arac\u0131l\u0131 gen terapilerinin bir risk ta\u015f\u0131d\u0131\u011f\u0131 bulundu ve bu da y\u00fcr\u00fcyemeyen DMD hastalar\u0131 i\u00e7in fayda-risk profilinin olumsuz olarak yeniden de\u011ferlendirilmesine neden oldu. \u2013 Daha Fazlas\u0131n\u0131 Okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\" target=\"_blank\" rel=\"noreferrer noopener\">Sarepta, Elevidys Gen Terapisi Tedavisinden Sonra 16 Ya\u015f\u0131ndaki \u00c7ocu\u011fun \u00d6l\u00fcm\u00fcn\u00fc Bildirdi<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Yeni doz k\u0131s\u0131tlamalar\u0131, herhangi bir ya\u015ftaki ayaktan DMD hastas\u0131n\u0131n tedavisini etkilememektedir ve ayaktan hasta pop\u00fclasyonunda fayda-risk oran\u0131 pozitif kalmaya devam etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0130ki \u00f6l\u00fcmc\u00fcl ALF vakas\u0131, bug\u00fcne kadar d\u00fcnya \u00e7ap\u0131nda Elevidys ile tedavi edilen yakla\u015f\u0131k 140 y\u00fcr\u00fcyemeyen hastadan, y\u00fcr\u00fcyemeyen hastalarda meydana geldi. \u0130lk \u00f6l\u00fcmc\u00fcl ALF vakas\u0131n\u0131n ard\u0131ndan, Avrupa d\u00fczenleyicileri Roche ve Sarepta&#039;dan Elevidys \u00e7al\u0131\u015fmalar\u0131 104 (NCT06241950), 302 (ENVOL, NCT06128564) ve 303 (ENVISION \u00c7al\u0131\u015fmas\u0131 303, NCT05310071) i\u00e7in ge\u00e7ici klinik beklemeler koymalar\u0131n\u0131 talep etti. Ge\u00e7ici klinik beklemeler hala y\u00fcr\u00fcrl\u00fcktedir. Avrupa d\u0131\u015f\u0131nda, ENVISION \u00e7al\u0131\u015fmas\u0131 i\u00e7in dozajlama derhal ge\u00e7erli olmak \u00fczere durdurulacakt\u0131r. Dozlama k\u0131s\u0131tlamalar\u0131, ticari y\u00fcr\u00fcyemeyen hastalar\u0131n gelecekteki dozajlamas\u0131 i\u00e7in de y\u00fcr\u00fcrl\u00fc\u011fe girecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">Elevidys Hakk\u0131nda S\u0131k\u00e7a Sorulan Sorular<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, has received a safety update from Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases. The company also discussed the actions it is taking to improve the safety profile in non-ambulatory patients. Following a second documented instance of acute [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3926,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[121,33,61,203,65],"class_list":["post-3925","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-gene-therapy","tag-roche","tag-sarepta"],"subtitle":"Sarepta, ticari ortamda ayakta tedavi edilemeyen hastalara inf\u00fczyon ama\u00e7l\u0131 ELEVIDYS sevkiyatlar\u0131n\u0131n, geli\u015ftirilmi\u015f rejim onaylan\u0131p y\u00fcr\u00fcrl\u00fc\u011fe girinceye kadar ask\u0131ya al\u0131nd\u0131\u011f\u0131n\u0131 bildiriyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3925","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=3925"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3925\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3926"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3925"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3925"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3925"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}