{"id":3768,"date":"2025-04-25T17:54:03","date_gmt":"2025-04-25T14:54:03","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3768"},"modified":"2025-06-21T13:46:46","modified_gmt":"2025-06-21T10:46:46","slug":"ema-recommends-eu-approval-for-duvyzat-givinostat-to-treat-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/ema-recommends-eu-approval-for-duvyzat-givinostat-to-treat-duchenne-muscular-dystrophy\/","title":{"rendered":"EMA, Duchenne Musk\u00fcler Distrofisini Tedavi Etmek \u0130\u00e7in Duvyzat'\u0131n (givinostat) AB Onay\u0131 Almas\u0131n\u0131 \u00d6nerdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avrupa \u0130la\u00e7 Ajans\u0131 (EMA), Duvyzat&#039;ye (givinostat) alt\u0131 ya\u015f\u0131ndan itibaren y\u00fcr\u00fcyebilen bireylerde Duchenne kas distrofisi (DMD) tedavisi i\u00e7in AB&#039;de ko\u015fullu pazarlama yetkisi verilmesini \u00f6nerdi. Duvyzat, kortikosteroid tedavisiyle birlikte oral yoldan verilen bir s\u00fcspansiyondur.<\/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=\"#is-duvyzat-a-treatment-for-dmd\">Duvyzat DMD \u0130\u00e7in Bir Tedavi midir?<\/a><\/li><li><a href=\"#is-duvyzat-safe\">Duvyzat G\u00fcvenli mi?<\/a><\/li><li><a href=\"#duvyzat-approved-in-europe\">Duvyzat Avrupa&#039;da Onayland\u0131<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"is-duvyzat-a-treatment-for-dmd\">Duvyzat DMD \u0130\u00e7in Bir Tedavi midir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD&#039;nin kesin bir tedavisi yoktur ve tedavi kortikosteroid tedavisine, kontrakt\u00fcrlerin \u00f6nlenmesine ve kalp ve solunum fonksiyonlar\u0131n\u0131n t\u0131bbi bak\u0131m\u0131na odaklan\u0131r.<a href=\"https:\/\/dmdwarrior.com\/tr\/duvyzat-givinostat-nonsteroidal-drug\/\" target=\"_blank\" data-type=\"post\" data-id=\"1462\" rel=\"noreferrer noopener\">Duvyzat (givinostat) nedir?<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"is-duvyzat-safe\">Duvyzat G\u00fcvenli mi?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duvyzat&#039;nin g\u00fcvenlik profili 179 hastadan al\u0131nan verilere dayanmaktad\u0131r. Givinostat ile tedavi edilen hastalarda en s\u0131k g\u00f6r\u00fclen olaylar ishal, kar\u0131n a\u011fr\u0131s\u0131, trombositopeni (kandaki trombositlerin d\u00fc\u015f\u00fck seviyeleri), kusma, hipertrigliseridemi (bir ya\u011f t\u00fcr\u00fc olan trigliseridlerin y\u00fcksek kan seviyeleri) ve ate\u015ftir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"duvyzat-approved-in-europe\">Duvyzat Avrupa&#039;da Onayland\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Duvyzat, kar\u015f\u0131lanmam\u0131\u015f t\u0131bbi bir ihtiyac\u0131 kar\u015f\u0131layan ila\u00e7lara erken eri\u015fimi kolayla\u015ft\u0131rmak i\u00e7in AB&#039;nin d\u00fczenleyici mekanizmalar\u0131ndan biri olan ko\u015fullu pazarlama yetkisi i\u00e7in \u00f6nerilir. Bu t\u00fcr bir onay, bir ilac\u0131n hastalara hemen sunulmas\u0131n\u0131n faydas\u0131, t\u00fcm verilerin hen\u00fcz mevcut olmamas\u0131 ger\u00e7e\u011finin i\u00e7erdi\u011fi riskten daha a\u011f\u0131r basarsa, Ajans\u0131n normalde beklenenden daha az eksiksiz veriyle bir ilac\u0131 pazarlama yetkisi i\u00e7in \u00f6nermesine olanak tan\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Duvyzat&#039;nin etkinli\u011fini do\u011frulamak amac\u0131yla, \u015firketten ayaktan DMD hastalar\u0131nda randomize, plasebo kontroll\u00fc bir \u00e7al\u0131\u015fma ve givinostat ile tedavi edilen alt\u0131 ya\u015f ve \u00fczeri DMD&#039;li \u00e7ocuklardan olu\u015fan bir hasta kay\u0131t defterinden elde edilen verilere dayal\u0131 bir ruhsatland\u0131rma sonras\u0131 etkililik ve g\u00fcvenlik \u00e7al\u0131\u015fmas\u0131 y\u00fcr\u00fctmesi talep edildi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CHMP taraf\u0131ndan kabul edilen g\u00f6r\u00fc\u015f, Duvyzat&#039;nin hasta eri\u015fimi yolunda bir ara ad\u0131md\u0131r. G\u00f6r\u00fc\u015f \u015fimdi AB \u00e7ap\u0131nda bir pazarlama yetkisi hakk\u0131nda bir karar\u0131n kabul\u00fc i\u00e7in Avrupa Komisyonu&#039;na g\u00f6nderilecektir. Bir pazarlama yetkisi verildikten sonra, fiyat ve geri \u00f6demeyle ilgili kararlar, bu ilac\u0131n o \u00fclkenin ulusal sa\u011fl\u0131k sistemi ba\u011flam\u0131ndaki potansiyel rol\u00fc\/kullan\u0131m\u0131 dikkate al\u0131narak her \u00dcye Devlet d\u00fczeyinde ger\u00e7ekle\u015fecektir.<\/p>","protected":false},"excerpt":{"rendered":"<p>The European Medicines Agency (EMA) has suggested that Duvyzat (givinostat) be given a conditional marketing authorization in the EU to treat Duchenne muscular dystrophy (DMD) in individuals who can walk starting at age six. Duvyzat is a suspension that is given orally in conjunction with corticosteroid therapy. Is Duvyzat a Treatment for DMD? There is [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3770,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[45],"tags":[46,91,213,47],"class_list":["post-3768","post","type-post","status-publish","format-standard","has-post-thumbnail","category-approved-therapies-for-duchenne-muscular-dystrophy","tag-duvyzat","tag-ema","tag-european-medicines-agency","tag-givinostat"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3768","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=3768"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3768\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3770"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3768"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3768"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3768"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}