{"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\/ar\/genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels\/","title":{"rendered":"\u0646\u062a\u0627\u0626\u062c \u062a\u062c\u0631\u0628\u0629 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a GNT0004 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0640 Genethon: \u0627\u0646\u062e\u0641\u0627\u0636 \u062f\u0627\u0626\u0645 \u0641\u064a \u0645\u0633\u062a\u0648\u064a\u0627\u062a CK \u0644\u0623\u0643\u062b\u0631 \u0645\u0646 75%"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u062a\u064f\u0638\u0647\u0631 \u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0648\u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0648\u0627\u0644\u062f\u064a\u0646\u0627\u0645\u064a\u0643\u064a\u0629 \u0627\u0644\u062f\u0648\u0627\u0626\u064a\u0629 \u062a\u062d\u0645\u0651\u0644\u0627\u064b \u062c\u064a\u062f\u0627\u064b \u0644\u062f\u0648\u0627\u0621 GNT0004 \u0627\u0644\u0645\u0631\u062a\u0628\u0637 \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0648\u0642\u0627\u0626\u064a \u0627\u0644\u0645\u0646\u0627\u0639\u064a \u0627\u0644\u0645\u0624\u0642\u062a\u060c \u0628\u0627\u0644\u0625\u0636\u0627\u0641\u0629 \u0625\u0644\u0649 \u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0645\u0646 \u062d\u064a\u062b \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0639\u0646 \u0645\u064a\u0643\u0631\u0648\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0648\u0627\u0646\u062e\u0641\u0627\u0636 \u0625\u0646\u0632\u064a\u0645 CPK\u060c \u0648\u0627\u0644\u0645\u0639\u0627\u064a\u064a\u0631 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 (NSAA\u060c \u0648\u0627\u0644\u0627\u062e\u062a\u0628\u0627\u0631\u0627\u062a \u0627\u0644\u0645\u0648\u0642\u0648\u062a\u0629). \u0623\u0638\u0647\u0631 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0641\u0639\u0627\u0644\u0629 \u062a\u062d\u0633\u0646\u0627\u064b \u0623\u0648 \u0627\u0633\u062a\u0642\u0631\u0627\u0631\u0627\u064b \u0645\u0637\u0648\u0644\u0627\u064b \u0641\u064a \u0627\u0644\u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u062d\u0631\u0643\u064a\u0629\u060c \u0648\u0627\u0646\u062e\u0641\u0627\u0636\u0627\u064b \u0645\u0644\u062d\u0648\u0638\u0627\u064b \u0648\u0645\u0633\u062a\u0645\u0631\u0627\u064b \u0641\u064a \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0625\u0646\u0632\u064a\u0645 \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 \u0643\u064a\u0646\u0627\u0632 (CPK)\u060c \u0648\u0647\u0648 \u0645\u0624\u0634\u0631 \u0631\u0626\u064a\u0633\u064a \u0644\u062a\u0644\u0641 \u0627\u0644\u0639\u0636\u0644\u0627\u062a.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0644\u0644\u062a\u062c\u0631\u0628\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0640 GNT0004<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u064a\u0624\u0643\u062f \u0645\u0646\u062a\u062c GNT0004 \u0641\u0639\u0627\u0644\u064a\u062a\u0647 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0641\u064a \u062a\u062b\u0628\u064a\u062a \u0627\u0644\u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u062d\u0631\u0643\u064a\u0629 \u0644\u062f\u0649 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0641\u0639\u0627\u0644\u0629 \u0644\u0645\u062f\u0629 \u062a\u0635\u0644 \u0625\u0644\u0649 \u0639\u0627\u0645\u064a\u0646.<\/li>\n\n\n\n<li>\u0627\u0646\u062e\u0641\u0627\u0636 \u0643\u0628\u064a\u0631 \u0648\u0645\u0633\u062a\u062f\u0627\u0645 \u0641\u064a \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0641\u0648\u0633\u0641\u0648\u0643\u064a\u0646\u0627\u0632 \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 (CPK)\u060c \u0648\u0647\u0648 \u0645\u0624\u0634\u0631 \u062d\u064a\u0648\u064a \u0644\u062a\u0644\u0641 \u0627\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0645\u0639 \u0627\u0646\u062e\u0641\u0627\u0636 \u0645\u062a\u0648\u0633\u0637 \u064a\u0632\u064a\u062f \u0639\u0646 75% \u0641\u064a 18 \u0634\u0647\u0631\u064b\u0627 \u0641\u064a \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u062b\u0644\u0627\u062b\u0629 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0641\u0639\u0627\u0644\u0629.<\/li>\n\n\n\n<li>\u0648\u0645\u0646 \u0627\u0644\u0645\u0642\u0631\u0631 \u062a\u0646\u0641\u064a\u0630 \u0645\u0631\u062d\u0644\u0629 \u0645\u062d\u0648\u0631\u064a\u0629 \u0641\u064a \u0645\u0646\u062a\u0635\u0641 \u0639\u0627\u0645 2025 \u0641\u064a \u0623\u0648\u0631\u0648\u0628\u0627 \u0648\u0627\u0644\u0648\u0644\u0627\u064a\u0627\u062a \u0627\u0644\u0645\u062a\u062d\u062f\u0629.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u0628\u0639\u062f \u0645\u0631\u0648\u0631 \u0639\u0627\u0645 \u0639\u0644\u0649 \u0627\u0644\u0639\u0644\u0627\u062c\u060c \u062a\u0638\u0647\u0631 \u0645\u0642\u0627\u0631\u0646\u0629 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u062b\u0644\u0627\u062b\u0629 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0641\u0639\u0627\u0644\u0629 \u0645\u0639 \u0645\u062c\u0645\u0648\u0639\u0629 \u0645\u0646 34 \u0645\u0631\u064a\u0636\u064b\u0627 \u063a\u064a\u0631 \u0645\u0639\u0627\u0644\u062c\u064a\u0646\u060c \u0645\u062a\u0637\u0627\u0628\u0642\u064a\u0646 \u062d\u0633\u0628 \u0627\u0644\u0639\u0645\u0631 \u0648\u064a\u062a\u0645 \u0645\u062a\u0627\u0628\u0639\u062a\u0647\u0645 \u0641\u064a \u0646\u0641\u0633 \u0627\u0644\u0645\u0631\u0627\u0643\u0632 \u0648\u0645\u0646 \u0642\u0628\u0644 \u0646\u0641\u0633 \u0627\u0644\u0645\u0645\u0627\u0631\u0633\u064a\u0646\u060c \u0641\u0631\u0642\u064b\u0627 \u0642\u062f\u0631\u0647 +4.7 \u0646\u0642\u0637\u0629 \u0641\u064a \u0627\u0644\u0646\u062a\u064a\u062c\u0629 \u0627\u0644\u062a\u064a \u062a\u0645 \u0627\u0644\u062d\u0635\u0648\u0644 \u0639\u0644\u064a\u0647\u0627 \u0628\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0645\u0642\u064a\u0627\u0633 \u0627\u0644\u062a\u0642\u064a\u064a\u0645 \u0627\u0644\u0633\u0631\u064a\u0631\u064a \u0627\u0644\u0645\u0639\u062a\u0631\u0641 \u0628\u0647 \u062f\u0648\u0644\u064a\u064b\u0627 NSAA \u0628\u064a\u0646 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u064a\u0646 \u0648\u063a\u064a\u0631 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u064a\u0646.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u0628\u0639\u062f 24 \u0634\u0647\u0631\u064b\u0627 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c\u060c \u062a\u062a\u0636\u0645\u0646 \u0627\u0644\u0645\u0644\u0627\u062d\u0638\u0627\u062a \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0645\u0627 \u064a\u0644\u064a:<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0628\u0627\u0644\u0646\u0633\u0628\u0629 \u0644\u0644\u0645\u0631\u064a\u0636\u064a\u0646 \u0627\u0644\u0644\u0630\u064a\u0646 \u0623\u0643\u0645\u0644\u0627 \u0639\u0627\u0645\u064a\u0646 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c\u060c \u0645\u0646 \u0628\u064a\u0646 \u062b\u0644\u0627\u062b\u0629 \u0645\u0631\u0636\u0649 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0641\u0639\u0627\u0644\u0629\u060c \u0623\u0638\u0647\u0631\u062a \u0627\u0644\u062a\u062c\u0631\u0628\u0629 \u0627\u0633\u062a\u0642\u0631\u0627\u0631\u064b\u0627 \u0641\u064a \u0627\u0644\u0648\u0638\u0627\u0626\u0641 \u0627\u0644\u062d\u0631\u0643\u064a\u0629 \u0627\u0644\u0645\u064f\u0642\u0627\u0633\u0629 \u0628\u0645\u0642\u064a\u0627\u0633 NSAA\u060c \u0628\u064a\u0646\u0645\u0627 \u0623\u0638\u0647\u0631 \u0627\u0644\u0645\u0631\u0636\u0649 \u063a\u064a\u0631 \u0627\u0644\u0645\u064f\u0639\u0627\u0644\u062c\u064a\u0646 \u0645\u0646 \u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u062a\u0627\u0631\u064a\u062e \u0627\u0644\u0637\u0628\u064a\u0639\u064a \u0627\u0644\u0645\u0648\u0627\u0632\u064a\u0629 \u0627\u0646\u062e\u0641\u0627\u0636\u064b\u0627 \u0645\u062a\u0648\u0633\u0637\u064b\u0627 \u0645\u0633\u062a\u0645\u0631\u064b\u0627 \u0648\u0645\u0644\u062d\u0648\u0638\u064b\u0627 \u0641\u064a NSAA. \u0628\u0627\u0644\u0646\u0633\u0628\u0629 \u0644\u0645\u0631\u064a\u0636 \u0645\u064f\u0639\u0627\u0644\u062c \u0648\u0627\u062d\u062f\u060c \u0633\u0645\u062d \u0627\u0644\u062a\u062d\u0633\u0646 \u0627\u0644\u0645\u0644\u062d\u0648\u0638 \u0628\u0627\u0644\u0648\u0635\u0648\u0644 \u0625\u0644\u0649 \u0627\u0644\u062d\u062f \u0627\u0644\u0623\u0642\u0635\u0649 \u0644\u0644\u062f\u0631\u062c\u0629 \u0648\u0647\u0648 34 \u0628\u0639\u062f 12 \u0634\u0647\u0631\u064b\u0627\u060c \u0648\u0627\u0644\u0630\u064a \u062a\u0645 \u062a\u0623\u0643\u064a\u062f\u0647 \u0628\u0639\u062f 24 \u0634\u0647\u0631\u064b\u0627 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c.<\/li>\n\n\n\n<li>\u0627\u0633\u062a\u0642\u0631\u0627\u0631 \u0627\u0646\u062e\u0641\u0627\u0636 CPK \u0628\u064a\u0646 50% \u064887% \u0641\u064a \u0627\u0644\u0645\u062a\u0648\u0633\u0637: &gt;75% \u0628\u0639\u062f 18 \u0634\u0647\u0631\u064b\u0627 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c (\u0628\u064a\u0627\u0646\u0627\u062a \u0645\u0646 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u062b\u0644\u0627\u062b\u0629 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0641\u0639\u0627\u0644\u0629)\u060c \u0648\u0645\u0633\u062a\u0645\u0631 (\u0645\u062a\u0627\u0628\u0639\u0629 \u062a\u0635\u0644 \u0625\u0644\u0649 24 \u0634\u0647\u0631\u064b\u0627 \u0644\u0644\u0645\u0631\u064a\u0636\u064a\u0646 \u0627\u0644\u0623\u0648\u0644\u064a\u0646 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0647\u0630\u0647 \u0627\u0644\u062c\u0631\u0639\u0629).<\/li>\n\n\n\n<li>\u062a\u0645 \u062a\u0623\u0643\u064a\u062f \u0645\u0644\u0641 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0627\u0644\u0645\u0637\u0645\u0626\u0646 \u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0628\u0639\u062f \u0639\u0627\u0645\u064a\u0646 \u0645\u0646 \u0627\u0644\u062d\u0642\u0646\u060c \u062f\u0648\u0646 \u062d\u062f\u0648\u062b \u0622\u062b\u0627\u0631 \u062c\u0627\u0646\u0628\u064a\u0629 \u062e\u0637\u064a\u0631\u0629 \u0639\u0646\u062f \u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0645\u062d\u062f\u062f\u0629\u060c \u0648\u0627\u0644\u062a\u064a \u0647\u064a \u0623\u0642\u0644 \u0628\u0634\u0643\u0644 \u0645\u0644\u062d\u0648\u0638 \u0645\u0646 \u062a\u0644\u0643 \u0627\u0644\u0645\u0633\u062a\u062e\u062f\u0645\u0629 \u0641\u064a \u0645\u0646\u062a\u062c\u0627\u062a \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u0623\u062e\u0631\u0649 \u0642\u064a\u062f \u0627\u0644\u062a\u0637\u0648\u064a\u0631 \u0644\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/ar\/genethon-gnt0004-gene-therapy-duchenne-muscular-dystrophy\/\">\u0643\u0644 \u0645\u0627 \u064a\u062c\u0628 \u0623\u0646 \u062a\u0639\u0631\u0641\u0647 \u0639\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Genethon GNT0004<\/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\/ar\/wp-json\/wp\/v2\/posts\/3866","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/comments?post=3866"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3866\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3868"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3866"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3866"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3866"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}