Duchenne Musk\u00fcler Distrofi (DMD) i\u00e7in Sarepta Therapeutics geninden t\u00fcretilen bir gen tedavisi olan Elevidys, art\u0131k ayakta tedavi g\u00f6ren hastalar i\u00e7in mevcut ve ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA), g\u00f6n\u00fcll\u00fc dondurman\u0131n kald\u0131r\u0131lmas\u0131n\u0131 \u00f6neriyor. FDA'nin soru\u015fturmas\u0131, gen tedavisi \u00fcr\u00fcn\u00fcn\u00fcn 8 ya\u015f\u0131ndaki \u00e7ocu\u011fun \u00f6l\u00fcm\u00fcyle hi\u00e7bir ilgisi olmad\u0131\u011f\u0131n\u0131 ortaya koydu.<\/p>\n\n\n\n
Ciddi g\u00fcvenlik endi\u015feleri ve \u00f6l\u00fcmlerin ortaya \u00e7\u0131kmas\u0131n\u0131n ard\u0131ndan bu y\u0131l\u0131n ba\u015flar\u0131nda ba\u015flayan Sarepta gen terapisinin \u00f6yk\u00fcs\u00fc, d\u00fczenleyici kurumlar\u0131n sert m\u00fcdahalesiyle yeni bir boyut kazand\u0131. FDA m\u00fcdahale etti ve \u00fc\u00e7\u00fcnc\u00fc bir hastan\u0131n (ayn\u0131 zamanda AAV vekt\u00f6r\u00fc kullanan uzuv-ku\u015fak kas distrofisi i\u00e7in ilac\u0131n Faz I \u00e7al\u0131\u015fmas\u0131na kat\u0131lan 51 ya\u015f\u0131ndaki bir erkek) \u00f6l\u00fcm\u00fc \u00fczerine, Sarepta'n\u0131n ayakta tedavi g\u00f6ren hastalara y\u00f6nelik t\u00fcm ABD tedavi teslimatlar\u0131n\u0131 durdurmas\u0131n\u0131 talep etti. FDA, Cuma g\u00fcn\u00fc ba\u015fka bir \u00f6l\u00fcm bildirilmesinin ard\u0131ndan bir ara\u015ft\u0131rma ba\u015flatt\u0131.<\/p>\n\n\n\n
Ancak Pazartesi \u00f6\u011fleden sonra yay\u0131nlanan k\u0131sa bir a\u00e7\u0131klamada, FDA g\u00f6n\u00fcll\u00fc bekleme s\u00fcresinin kald\u0131r\u0131lmas\u0131n\u0131 ve ayakta tedavi g\u00f6ren ki\u015filerin Elevidys almaya devam etmelerine izin verilmesini \u00f6nerdi. Sarepta ve Brezilya yetkilileri, sekiz ya\u015f\u0131ndaki Brezilyal\u0131 hastan\u0131n son \u00f6l\u00fcm\u00fcn\u00fcn gen tedavisi \u00fcr\u00fcn\u00fcnden ziyade \u015fiddetli bir grip n\u00f6betinden kaynakland\u0131\u011f\u0131 konusunda kurumla ayn\u0131 fikirdeydi. Bu enfeksiyon, ba\u011f\u0131\u015f\u0131kl\u0131k sisteminin bask\u0131lanmas\u0131yla daha da k\u00f6t\u00fcle\u015fiyor. \u2013 Devam\u0131n\u0131 Oku: FDA, Elevidys Alan 8 Ya\u015f\u0131ndaki Brezilyal\u0131 \u00c7ocu\u011fun \u00d6l\u00fcm\u00fcn\u00fc Ara\u015ft\u0131r\u0131yor<\/a> \u2013<\/p>\n\n\n\n Elevidys ile ba\u011flant\u0131l\u0131 iki \u00f6l\u00fcmden sonra FDA'nin hen\u00fcz herhangi bir \u00f6neri yay\u0131nlamad\u0131\u011f\u0131, y\u00fcr\u00fcyemeyen hastalar i\u00e7in g\u00f6n\u00fcll\u00fc tutma hala y\u00fcr\u00fcrl\u00fcktedir.<\/p>","protected":false},"excerpt":{"rendered":" Elevidys, a gene therapy for Duchenne Muscular Dystrophy (DMD) from Sarepta Therapeutics, is now available to ambulatory patients, and the U.S. Food and Drug Administration is suggesting that the voluntary hold be lifted. The FDA’s inquiry found that the gene therapy product had nothing to do with the 8-year-old boy’s death. The story of Sarepta’s […]<\/p>\n","protected":false},"author":1,"featured_media":4239,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[33,179,88,61,203,65],"class_list":["post-4237","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevidys","tag-elevidys-gene-therapy","tag-fda","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4237","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=4237"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4237\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4239"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4237"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4237"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4237"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}