{"id":3866,"date":"2025-05-19T13:43:11","date_gmt":"2025-05-19T10:43:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3866"},"modified":"2025-05-19T13:43:16","modified_gmt":"2025-05-19T10:43:16","slug":"genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels\/","title":{"rendered":"Genethon, GNT0004 Gen Terapisi Klinik Denemesinin Sonu\u00e7lar\u0131n\u0131 Bildiriyor: 75%&#039;den Fazla CK D\u00fczeylerinde Kal\u0131c\u0131 Azalma"},"content":{"rendered":"<p class=\"wp-block-paragraph\">G\u00fcvenlik, etkililik ve farmakodinamik sonu\u00e7lar, ge\u00e7ici imm\u00fcnolojik profilaktik tedaviyle ili\u015fkili GNT0004&#039;\u00fcn iyi tolerans\u0131n\u0131 ve mikrodistrofin ekspresyonu, CPK azalmas\u0131 ve klinik kriterler (NSAA, zamanl\u0131 testler) a\u00e7\u0131s\u0131ndan etkililik verilerini g\u00f6stermektedir. Etkili dozda tedavi edilen hastalar, motor fonksiyonlar\u0131nda uzun s\u00fcreli iyile\u015fme veya stabilizasyon ve kas hasar\u0131n\u0131n \u00f6nemli bir belirteci olan kreatin kinaz (CPK) seviyelerinde \u00f6nemli kal\u0131c\u0131 azalma g\u00f6stermektedir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">GNT0004 Klinik Denemesinin Temel Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>GNT0004 \u00fcr\u00fcn\u00fc, etkili dozda 2 y\u0131la kadar tedavi edilen hastalarda motor fonksiyonlar\u0131n stabilizasyonu ile klinik etkinli\u011fini do\u011frulamaktad\u0131r.<\/li>\n\n\n\n<li>Etkili dozda tedavi edilen 3 hastada, 18 ayda kas hasar\u0131n\u0131n biyobelirteci olan kreatin fosfokinaz (CPK) d\u00fczeylerinde anlaml\u0131 d\u00fczeyde kal\u0131c\u0131 azalma ve ortalama 75%&#039;nin \u00fczerinde azalma g\u00f6zlendi.<\/li>\n\n\n\n<li>Avrupa ve ABD&#039;de kritik a\u015faman\u0131n 2025 ortalar\u0131nda ger\u00e7ekle\u015fmesi planlan\u0131yor.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Tedaviden bir y\u0131l sonra, etkili dozda tedavi edilen \u00fc\u00e7 hasta ile ayn\u0131 merkezlerde ve ayn\u0131 uygulay\u0131c\u0131lar taraf\u0131ndan takip edilen, ya\u015fa g\u00f6re e\u015fle\u015ftirilmi\u015f, tedavi edilmeyen 34 hastadan olu\u015fan bir grup kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda, uluslararas\u0131 kabul g\u00f6rm\u00fc\u015f klinik de\u011ferlendirme \u00f6l\u00e7e\u011fi NSAA kullan\u0131larak tedavi edilen ve tedavi edilmeyen hastalar aras\u0131nda elde edilen puanda +4,7 puanl\u0131k bir fark oldu\u011fu g\u00f6r\u00fcld\u00fc.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Tedaviden 24 ay sonra, temel g\u00f6zlemler \u015funlard\u0131r:<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Etkili dozda tedavi edilen 3 hastadan tedavi sonras\u0131 2 y\u0131la ula\u015fan 2 hasta i\u00e7in, \u00e7al\u0131\u015fma NSAA \u00f6l\u00e7e\u011fiyle \u00f6l\u00e7\u00fclen motor fonksiyonlar\u0131n stabilizasyonunu g\u00f6sterirken, paralel do\u011fal tarih \u00e7al\u0131\u015fmas\u0131ndan tedavi edilmeyen hastalar NSAA&#039;da s\u00fcrekli ve anlaml\u0131 bir ortalama d\u00fc\u015f\u00fc\u015f g\u00f6sterdi. Tedavi edilen bir hasta i\u00e7in, g\u00f6zlenen iyile\u015fme 12 ayda 34&#039;l\u00fck maksimum puana ula\u015fmay\u0131 sa\u011flad\u0131 ve bu, tedavi sonras\u0131 24 ayda do\u011fruland\u0131.<\/li>\n\n\n\n<li>50% ile 87% aras\u0131ndaki CPK azalmas\u0131n\u0131n ortalama olarak sabitlenmesi: tedavi sonras\u0131 18 ayda &gt;75% (etkili dozda tedavi edilen 3 hastadan elde edilen veriler) ve kal\u0131c\u0131 (bu dozda tedavi edilen ilk iki hasta i\u00e7in 24 aya kadar takip).<\/li>\n\n\n\n<li>Gen tedavisi ilac\u0131n\u0131n g\u00fcven verici g\u00fcvenlik profili, enjeksiyondan iki y\u0131l sonra do\u011fruland\u0131 ve se\u00e7ilen dozda ciddi yan etkiler g\u00f6r\u00fclmedi; bu doz, Duchenne Musk\u00fcler Distrofi i\u00e7in geli\u015ftirilmekte olan di\u011fer gen tedavisi \u00fcr\u00fcnlerinde kullan\u0131lan dozdan belirgin \u015fekilde daha d\u00fc\u015f\u00fckt\u00fcr.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/genethon-gnt0004-gene-therapy-duchenne-muscular-dystrophy\/\">Genethon&#039;nin Gen Terapisi GNT0004 Hakk\u0131nda Bilmeniz Gereken Her \u015eey<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of microdystrophin expression, CPK reduction, and clinical criteria (NSAA, timed tests). Patients treated at the effective dose show prolonged improvement or stabilization of motor functions and significant persistent reduction in creatine kinase [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3868,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[117,66,342,61,105,221,220],"class_list":["post-3866","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dmd","tag-duchenne-muscular-dystrophy","tag-frederic-revah","tag-gene-therapy","tag-genethon","tag-gnt-0004-2","tag-gnt0004"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3866","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=3866"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3866\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3868"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3866"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3866"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3866"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}