{"id":3218,"date":"2025-03-17T10:34:47","date_gmt":"2025-03-17T07:34:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3218"},"modified":"2025-03-17T10:39:51","modified_gmt":"2025-03-17T07:39:51","slug":"dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics \u064a\u0639\u0644\u0646 \u0639\u0646 \u062a\u062d\u0633\u0646 \u0648\u0638\u064a\u0641\u064a \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642 \u0648\u0645\u0633\u062a\u062f\u0627\u0645 \u062e\u0644\u0627\u0644 18 \u0634\u0647\u0631\u064b\u0627 \u0645\u0646 \u0627\u0644\u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u0623\u0648\u0644\u0649 \u0648\u0627\u0644\u062b\u0627\u0646\u064a\u0629 \u0645\u0646 \u062a\u062c\u0631\u0628\u0629 DELIVER \u0644\u0640 DYNE-251 \u0641\u064a \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0623\u0639\u0644\u0646\u062a \u0627\u0644\u064a\u0648\u0645 \u0634\u0631\u0643\u0629 Dyne Therapeutics\u060c \u0648\u0647\u064a \u0634\u0631\u0643\u0629 \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u062a\u0631\u0643\u0632 \u0639\u0644\u0649 \u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062a\u064a \u062a\u063a\u064a\u0631 \u062d\u064a\u0627\u0629 \u0627\u0644\u0623\u0634\u062e\u0627\u0635 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u0623\u0645\u0631\u0627\u0636 \u0639\u0635\u0628\u064a\u0629 \u0639\u0636\u0644\u064a\u0629 \u0648\u0631\u0627\u062b\u064a\u0629\u060c \u0639\u0646 \u0628\u064a\u0627\u0646\u0627\u062a \u0633\u0631\u064a\u0631\u064a\u0629 \u0637\u0648\u064a\u0644\u0629 \u0627\u0644\u0623\u0645\u062f \u062c\u062f\u064a\u062f\u0629 \u0645\u0646 \u062a\u062c\u0631\u0628\u062a\u0647\u0627 \u0627\u0644\u062c\u0627\u0631\u064a\u0629 \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2 DELIVER \u0644\u0640 DYNE-251 \u0648\u0627\u0644\u062a\u064a \u0623\u0638\u0647\u0631\u062a \u062a\u062d\u0633\u0646\u064b\u0627 \u0648\u0638\u064a\u0641\u064a\u064b\u0627 \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642 \u0648\u0645\u0633\u062a\u062f\u0627\u0645\u064b\u0627 \u0639\u0646\u062f \u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u062a\u0633\u062c\u064a\u0644\u064a\u0629 \u0627\u0644\u0645\u062d\u062f\u062f\u0629 20 \u0645\u062c\u0645 \/ \u0643\u062c\u0645 Q4W (\u062c\u0631\u0639\u0629 PMO \u0627\u0644\u062a\u0642\u0631\u064a\u0628\u064a\u0629).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u0642\u0648\u0645 \u062a\u062c\u0631\u0628\u0629 DELIVER \u0628\u062a\u0642\u064a\u064a\u0645 DYNE-251 \u0641\u064a \u0627\u0644\u0623\u0641\u0631\u0627\u062f \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD) \u0648\u0627\u0644\u0630\u064a\u0646 \u064a\u0645\u0643\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51\u060c \u0648\u0633\u064a\u062a\u0645 \u062a\u0642\u062f\u064a\u0645 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0645\u062d\u062f\u062b\u0629 \u0645\u0646 \u0627\u0644\u062a\u062c\u0631\u0628\u0629 \u0647\u0630\u0627 \u0627\u0644\u0623\u0633\u0628\u0648\u0639 \u0641\u064a \u0627\u0644\u0645\u0624\u062a\u0645\u0631 \u0627\u0644\u0633\u0631\u064a\u0631\u064a \u0648\u0627\u0644\u0639\u0644\u0645\u064a \u0644\u062c\u0645\u0639\u064a\u0629 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a (MDA) \u0644\u0639\u0627\u0645 2025.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u062a\u062a\u0636\u0645\u0646 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0645\u0646 \u0639\u0631\u0636 \u062a\u062c\u0631\u0628\u0629 DELIVER \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2 \u0645\u0627 \u064a\u0644\u064a:<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0648\u0638\u064a\u0641\u0629:<\/strong> \u0644\u0648\u062d\u0638\u062a \u062a\u062d\u0633\u064a\u0646\u0627\u062a \u0645\u0644\u062d\u0648\u0638\u0629 \u0648\u0645\u0633\u062a\u062f\u0627\u0645\u0629 \u0645\u0646 \u062e\u0637 \u0627\u0644\u0623\u0633\u0627\u0633 \u0641\u064a \u0646\u0642\u0627\u0637 \u0646\u0647\u0627\u064a\u0629 \u0648\u0638\u064a\u0641\u064a\u0629 \u0645\u062a\u0639\u062f\u062f\u0629 \u0641\u064a \u0643\u0644\u064d\u0651 \u0645\u0646 \u0645\u062c\u0645\u0648\u0639\u062a\u064a \u062c\u0631\u0639\u0629 20 \u0645\u0644\u063a\/\u0643\u063a (\u062c\u0631\u0639\u0629 \u0627\u0644\u062a\u0633\u062c\u064a\u0644 \u0627\u0644\u0645\u062e\u062a\u0627\u0631\u0629) \u064810 \u0645\u0644\u063a\/\u0643\u063a1 DYNE-251 \u0631\u0628\u0639 \u0627\u0644\u0633\u0646\u0648\u064a\u0629\u060c \u0639\u0644\u0649 \u0645\u062f\u0627\u0631 12 \u064818 \u0634\u0647\u0631\u064b\u0627 \u0639\u0644\u0649 \u0627\u0644\u062a\u0648\u0627\u0644\u064a. \u0648\u0634\u0645\u0644\u062a \u0627\u0644\u062a\u0642\u064a\u064a\u0645\u0627\u062a \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0645\u0642\u064a\u0627\u0633 \u0633\u0631\u0639\u0629 \u0627\u0644\u062e\u0637\u0648\u0629 \u0627\u0644\u0645\u0626\u0648\u064a \u0627\u0644\u062e\u0627\u0645\u0633 \u0648\u0627\u0644\u062a\u0633\u0639\u064a\u0646 (SV95C)\u060c \u0648\u062a\u0642\u064a\u064a\u0645 \u062d\u0631\u0643\u0629 \u0646\u062c\u0645 \u0627\u0644\u0634\u0645\u0627\u0644 (NSAA)\u060c \u0648\u0632\u0645\u0646 \u0627\u0644\u0645\u0634\u064a\/\u0627\u0644\u062c\u0631\u064a 10 \u0623\u0645\u062a\u0627\u0631 (10-MWR)\u060c \u0648\u0632\u0645\u0646 \u0627\u0644\u0646\u0647\u0648\u0636 \u0645\u0646 \u0627\u0644\u0623\u0631\u0636.\n<ul class=\"wp-block-list\">\n<li>\u0628\u062f\u0621\u064b\u0627 \u0645\u0646 \u0646\u0642\u0637\u0629 \u0632\u0645\u0646\u064a\u0629 \u0645\u062f\u062a\u0647\u0627 6 \u0623\u0634\u0647\u0631\u060c \u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u062a\u063a\u064a\u064a\u0631 \u0641\u064a SV95C \u0645\u0646 \u062e\u0637 \u0627\u0644\u0623\u0633\u0627\u0633 \u0627\u0644\u0630\u064a \u0644\u0648\u062d\u0638 \u0641\u064a \u0643\u0644 \u0645\u0646 \u0645\u062c\u0645\u0648\u0639\u062a\u064a 10 \u0645\u062c\u0645 \/ \u0643\u062c\u0645 \u0648 20 \u0645\u062c\u0645 \/ \u0643\u062c\u0645 \u0645\u0646 DELIVER \u0627\u0644\u0641\u0631\u0642 \u0627\u0644\u0633\u0631\u064a\u0631\u064a \u0627\u0644\u0645\u0647\u0645 \u0627\u0644\u0623\u062f\u0646\u0649 \u0627\u0644\u0645\u0642\u062a\u0631\u062d (MCID) \u0627\u0644\u0645\u0646\u0634\u0648\u0631.<\/li>\n\n\n\n<li>SV95C \u0647\u0648 \u0645\u0642\u064a\u0627\u0633 \u0631\u0642\u0645\u064a \u0645\u0648\u0636\u0648\u0639\u064a \u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0623\u062f\u0627\u0621 \u0627\u0644\u062d\u0631\u0643\u064a \u0641\u064a \u0627\u0644\u0628\u064a\u0626\u0629 \u0627\u0644\u064a\u0648\u0645\u064a\u0629 \u0627\u0644\u0637\u0628\u064a\u0639\u064a\u0629 \u0644\u0644\u0645\u0631\u0636\u0649 \u0648\u064a\u062a\u0645 \u0642\u0628\u0648\u0644\u0647 \u0643\u0646\u0642\u0637\u0629 \u0646\u0647\u0627\u064a\u0629 \u0623\u0633\u0627\u0633\u064a\u0629 \u0644\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0641\u064a \u0623\u0648\u0631\u0648\u0628\u0627.<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0639\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646:<\/strong> \u0643\u0645\u0627 \u0630\u064f\u0643\u0631 \u0633\u0627\u0628\u0642\u064b\u0627\u060c \u0623\u0638\u0647\u0631 DYNE-251 \u062a\u0639\u0628\u064a\u0631\u064b\u0627 \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642 \u0644\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0637\u0648\u0644 \u0634\u0628\u0647 \u0643\u0627\u0645\u0644\u060c \u0643\u0645\u0627 \u062a\u0645 \u0642\u064a\u0627\u0633\u0647 \u0628\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0644\u0637\u062e\u0629 \u0648\u064a\u0633\u062a\u0631\u0646 \u0644\u062f\u0649 \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0627\u0644\u0630\u064a\u0646 \u064a\u064f\u062d\u062a\u0645\u0644 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. \u0628\u0639\u062f \u0633\u062a\u0629 \u0623\u0634\u0647\u0631\u060c \u0628\u0644\u063a \u0645\u062a\u0648\u0633\u0637 \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0627\u0644\u0645\u0637\u0644\u0642 \u0644\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0644\u062f\u0649 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u062c\u0631\u0639\u0629 20 \u0645\u0644\u063a\/\u0643\u063a \u0645\u0646 DYNE-251 \u0643\u0644 \u0623\u0631\u0628\u0639\u0629 \u0623\u0633\u0627\u0628\u064a\u0639 8.72%\u060c \u0648\u0647\u0648 \u0645\u0639\u062f\u0644 \u0637\u0628\u064a\u0639\u064a (\u0645\u064f\u0639\u062f\u0651\u0644 \u0644\u0645\u062d\u062a\u0648\u0649 \u0627\u0644\u0639\u0636\u0644\u0627\u062a). \u0623\u0643\u062f\u062a \u0634\u0631\u0643\u0629 \u062f\u0627\u064a\u0646 \u0623\u0646 \u0633\u0627\u0628\u0642\u0629 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u063a\u0630\u0627\u0621 \u0648\u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u0623\u0645\u0631\u064a\u0643\u064a\u0629 \u0644\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0645\u0624\u0634\u0631 \u062d\u064a\u0648\u064a \u0628\u062f\u064a\u0644 \u0644\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0627\u0644\u0633\u0631\u064a\u0639\u0629 \u0644\u0627 \u062a\u0632\u0627\u0644 \u0642\u0627\u0626\u0645\u0629.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0648\u0627\u0644\u062a\u062d\u0645\u0644:<\/strong> \u0623\u0638\u0647\u0631 DYNE-251 \u0645\u0633\u062a\u0648\u0649 \u0633\u0644\u0627\u0645\u0629 \u0625\u064a\u062c\u0627\u0628\u064a\u064b\u0627 \u0628\u0646\u0627\u0621\u064b \u0639\u0644\u0649 54 \u0645\u0634\u0627\u0631\u0643\u064b\u0627 \u0645\u0633\u062c\u0644\u064a\u0646 \u0641\u064a \u062a\u062c\u0631\u0628\u0629 DELIVER. \u0645\u0646\u0630 \u0627\u0644\u062a\u062d\u062f\u064a\u062b \u0627\u0644\u0633\u0627\u0628\u0642 \u0627\u0644\u0635\u0627\u062f\u0631 \u0641\u064a 21 \u0646\u0648\u0641\u0645\u0628\u0631 2024\u060c \u0638\u0644 \u0645\u0633\u062a\u0648\u0649 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u062f\u0648\u0646 \u062a\u063a\u064a\u064a\u0631\u060c \u0648\u0644\u0645 \u062a\u064f\u0644\u0627\u062d\u0638 \u0623\u064a \u0622\u062b\u0627\u0631 \u062c\u0627\u0646\u0628\u064a\u0629 \u062e\u0637\u064a\u0631\u0629 \u062c\u062f\u064a\u062f\u0629 \u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0639\u0644\u0627\u062c.\n<ul class=\"wp-block-list\">\n<li>\u062a\u0645 \u0625\u0639\u0637\u0627\u0621 970 \u062c\u0631\u0639\u0629 \u0645\u0646 \u0639\u0642\u0627\u0631 \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u062d\u062a\u0649 \u0627\u0644\u0622\u0646 \u0639\u0644\u0649 \u0645\u062f\u0649 \u0641\u062a\u0631\u0629 77.1 \u0633\u0646\u0629 \u0645\u0646 \u0627\u0644\u0645\u062a\u0627\u0628\u0639\u0629 \u0644\u0644\u0645\u0631\u0636\u0649\u060c \u0645\u0639 \u0645\u062a\u0627\u0628\u0639\u0629 \u0628\u0639\u0636 \u0627\u0644\u0645\u0631\u0636\u0649 \u0644\u0645\u062f\u0629 \u062a\u0635\u0644 \u0625\u0644\u0649 \u062d\u0648\u0627\u0644\u064a 2.5 \u0633\u0646\u0629.<\/li>\n\n\n\n<li>\u062a\u0645 \u062d\u062a\u0649 \u0627\u0644\u0622\u0646 \u0625\u0639\u0637\u0627\u0621 546 \u062c\u0631\u0639\u0629 \u0645\u0646 \u0639\u0642\u0627\u0631 \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0628\u0645\u0633\u062a\u0648\u0649 \u062c\u0631\u0639\u0629 20 \u0645\u062c\u0645 \/ \u0643\u062c\u0645.<\/li>\n<\/ul>\n<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics, a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data from its ongoing Phase 1\/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg\/kg Q4W (approximate PMO dose). The DELIVER trial is [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3221,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194],"class_list":["post-3218","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3218","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=3218"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3218\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3221"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3218"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3218"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3218"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}