{"id":5484,"date":"2026-01-26T16:38:21","date_gmt":"2026-01-26T13:38:21","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5484"},"modified":"2026-01-26T23:15:59","modified_gmt":"2026-01-26T20:15:59","slug":"sarepta-3-year-topline-data-of-elevidys-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/sarepta-3-year-topline-data-of-elevidys-gene-therapy\/","title":{"rendered":"\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 \u0633\u0627\u0631\u064a\u0628\u062a\u0627 \u0639\u0646 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0644\u0645\u062f\u0629 3 \u0633\u0646\u0648\u0627\u062a \u0644\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a ELEVIDYS \u0644\u0644\u0623\u0641\u0631\u0627\u062f \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a."},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 Sarepta \u0639\u0646 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0644\u0645\u062f\u0629 3 \u0633\u0646\u0648\u0627\u062a \u0645\u0646 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0641\u064a \u0627\u0644\u062c\u0632\u0621 \u0627\u0644\u0623\u0648\u0644 \u0645\u0646 \u062f\u0631\u0627\u0633\u0629 EMBARK (\u0627\u0644\u062f\u0631\u0627\u0633\u0629 9001-301)\u060c \u0648\u0647\u064a \u062f\u0631\u0627\u0633\u0629 \u0639\u0627\u0644\u0645\u064a\u0629 \u0639\u0634\u0648\u0627\u0626\u064a\u0629 \u062e\u0627\u0636\u0639\u0629 \u0644\u0644\u062a\u062d\u0643\u0645 \u0627\u0644\u0648\u0647\u0645\u064a \u0645\u0646 \u0627\u0644\u0645\u0631\u062d\u0644\u0629 3 \u0644\u062a\u0642\u064a\u064a\u0645 ELEVIDYS (delandistrogene moxeparvovec-rokl) \u0644\u062f\u0649 \u0627\u0644\u0623\u0641\u0631\u0627\u062f \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0648\u0627\u0644\u0630\u064a\u0646 \u062a\u062a\u0631\u0627\u0648\u062d \u0623\u0639\u0645\u0627\u0631\u0647\u0645 \u0628\u064a\u0646 \u0623\u0631\u0628\u0639 \u0648\u0633\u0628\u0639 \u0633\u0646\u0648\u0627\u062a \u0648\u0642\u062a \u0627\u0644\u0639\u0644\u0627\u062c.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0644\u0645 \u064a\u062a\u0645 \u0627\u0644\u0643\u0634\u0641 \u0639\u0646 \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 \u0643\u064a\u0646\u0627\u0632\u060c \u0648\u0647\u0648 \u0627\u0644\u0645\u0624\u0634\u0631 \u0627\u0644\u062d\u064a\u0648\u064a \u0627\u0644\u0631\u0626\u064a\u0633\u064a \u0644\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u0636\u0645\u0646 \u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0633\u0646\u0648\u0627\u062a \u0627\u0644\u062b\u0644\u0627\u062b.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u0641\u064a \u0645\u062a\u0648\u0633\u0637 \u0639\u0645\u0631 9 \u0633\u0646\u0648\u0627\u062a\u060c \u062d\u0642\u0642 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0640 ELEVIDYS \u0645\u062a\u0648\u0633\u0637 \u062f\u0631\u062c\u0627\u062a \u062a\u0642\u064a\u064a\u0645 \u0627\u0644\u0645\u0634\u064a \u0641\u064a \u0646\u0648\u0631\u062b \u0633\u062a\u0627\u0631 (NSAA) \u0623\u0639\u0644\u0649 \u0645\u0646 \u062e\u0637 \u0627\u0644\u0623\u0633\u0627\u0633 \u0628\u0639\u062f \u062b\u0644\u0627\u062b \u0633\u0646\u0648\u0627\u062a \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c (\u0646 = 52).<\/li>\n\n\n\n<li>\u0623\u0638\u0647\u0631 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a ELEVIDYS \u0627\u0646\u062e\u0641\u0627\u0636\u064b\u0627 \u0628\u0646\u0633\u0628\u0629 70% \u0623\u0648 \u0623\u0643\u062b\u0631 \u0641\u064a \u0645\u0639\u062f\u0644 \u0627\u0644\u062a\u062f\u0647\u0648\u0631 \u0645\u0642\u0627\u0631\u0646\u0629\u064b \u0628\u0645\u062c\u0645\u0648\u0639\u0629 \u0627\u0644\u062a\u062d\u0643\u0645 \u0627\u0644\u062e\u0627\u0631\u062c\u064a\u0629 \u0627\u0644\u0645\u0631\u062c\u062d\u0629 \u0628\u0627\u0644\u0627\u0633\u062a\u0639\u062f\u0627\u062f\u060c \u0648\u0630\u0644\u0643 \u0648\u0641\u0642\u064b\u0627 \u0644\u0642\u064a\u0627\u0633 &quot;\u0632\u0645\u0646 \u0627\u0644\u0646\u0647\u0648\u0636&quot; (TTR) \u0648\u0627\u062e\u062a\u0628\u0627\u0631 \u0627\u0644\u0645\u0634\u064a\/\u0627\u0644\u062c\u0631\u064a \u0644\u0645\u0633\u0627\u0641\u0629 10 \u0623\u0645\u062a\u0627\u0631 (10MWR). \u0648\u0623\u0638\u0647\u0631 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0640 ELEVIDYS \u062a\u062d\u0633\u0646\u064b\u0627 \u0645\u0644\u062d\u0648\u0638\u064b\u0627 \u0641\u064a \u0641\u0639\u0627\u0644\u064a\u0629 \u0627\u0644\u0639\u0644\u0627\u062c \u0645\u0639 \u0645\u0631\u0648\u0631 \u0627\u0644\u0648\u0642\u062a\u060c \u062d\u064a\u062b \u0627\u062a\u0633\u0639\u062a \u0627\u0644\u0641\u062c\u0648\u0629 \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0645\u0642\u0627\u0631\u0646\u0629\u064b \u0628\u0645\u062c\u0645\u0648\u0639\u0629 \u0627\u0644\u062a\u062d\u0643\u0645 \u0627\u0644\u062e\u0627\u0631\u062c\u064a\u0629 \u0628\u0634\u0643\u0644 \u0645\u0644\u062d\u0648\u0638 \u0628\u064a\u0646 \u0627\u0644\u0633\u0646\u0629 \u0627\u0644\u062b\u0627\u0646\u064a\u0629 \u0648\u0627\u0644\u0633\u0646\u0629 \u0627\u0644\u062b\u0627\u0644\u062b\u0629.<\/li>\n\n\n\n<li>\u0644\u0645 \u062a\u064f\u0644\u0627\u062d\u0638 \u0623\u064a \u0625\u0634\u0627\u0631\u0627\u062a \u0633\u0644\u0627\u0645\u0629 \u062c\u062f\u064a\u062f\u0629 \u0645\u062a\u0639\u0644\u0642\u0629 \u0628\u0627\u0644\u0639\u0644\u0627\u062c\u060c \u0648\u0647\u0648 \u0645\u0627 \u064a\u062a\u0648\u0627\u0641\u0642 \u0645\u0639 \u0645\u0644\u0641 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0627\u0644\u0642\u0627\u0628\u0644 \u0644\u0644\u062a\u062d\u0643\u0645 \u0627\u0644\u0630\u064a \u0644\u0648\u062d\u0638 \u0645\u0639 \u062f\u0648\u0627\u0621 ELEVIDYS \u0644\u062f\u0649 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u062e\u0627\u0631\u062c\u064a\u064a\u0646 \u062d\u062a\u0649 \u0627\u0644\u0622\u0646<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/ar\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u062a\u0642\u064a\u064a\u0645 \u0646\u0648\u0631\u062b \u0633\u062a\u0627\u0631 \u0644\u0644\u0639\u064a\u0627\u062f\u0627\u062a \u0627\u0644\u062e\u0627\u0631\u062c\u064a\u0629 (NSAA) \u0641\u064a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"778\" height=\"118\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png\" alt=\"\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 \u0633\u0627\u0631\u064a\u0628\u062a\u0627 \u0639\u0646 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0644\u0645\u062f\u0629 3 \u0633\u0646\u0648\u0627\u062a \u0644\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a ELEVIDYS \u0644\u0644\u0623\u0641\u0631\u0627\u062f \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a.\" class=\"wp-image-5496\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png 778w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-300x46.png 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-768x116.png 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-18x3.png 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-150x23.png 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-696x106.png 696w\" sizes=\"auto, (max-width: 778px) 100vw, 778px\" \/><\/figure>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td><strong>\u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629<\/strong><\/td><td><strong>\u0641\u0631\u0642 \u0627\u0644\u062a\u063a\u064a\u0631 \u0641\u064a LSM \u0645\u0642\u0627\u0628\u0644 EC<\/strong><\/td><td><strong>\u0642\u064a\u0645\u0629 p<\/strong><\/td><\/tr><tr><td>\u0648\u0643\u0627\u0644\u0629 \u0627\u0644\u0623\u0645\u0646 \u0627\u0644\u0642\u0648\u0645\u064a<\/td><td>+4.39 \u0646\u0642\u0637\u0629 (\u062a\u062d\u0633\u0646)<\/td><td>p=0.0002<\/td><\/tr><tr><td>TTR<\/td><td>-6.05 \u062b\u0627\u0646\u064a\u0629 (\u062a\u062d\u0633\u0646)<\/td><td>p&lt;0.0001<\/td><\/tr><tr><td>10MWR<\/td><td>-2.70 \u062b\u0627\u0646\u064a\u0629 (\u062a\u062d\u0633\u0646)<\/td><td>p=0.0039<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">\u0641\u064a 26 \u064a\u0646\u0627\u064a\u0631 2026\u060c \u0628\u062f\u0623 \u062a\u062f\u0627\u0648\u0644 \u0623\u0633\u0647\u0645 \u0634\u0631\u0643\u0629 \u0633\u0627\u0631\u064a\u0628\u062a\u0627 \u0628\u0633\u0639\u0631 22 \u062f\u0648\u0644\u0627\u0631\u064b\u0627 \u0623\u0645\u0631\u064a\u0643\u064a\u064b\u0627\u060c \u062b\u0645 \u0627\u0631\u062a\u0641\u0639 \u0625\u0644\u0649 24.68 \u062f\u0648\u0644\u0627\u0631\u064b\u0627 \u0623\u0645\u0631\u064a\u0643\u064a\u064b\u0627 \u0628\u0639\u062f \u0646\u0634\u0631 \u0628\u064a\u0627\u0646\u0627\u062a \u062b\u0644\u0627\u062b \u0633\u0646\u0648\u0627\u062a \u062d\u0648\u0644 \u0639\u0644\u0627\u062c\u0647\u0627 \u0627\u0644\u062c\u064a\u0646\u064a Elevidys. \u0625\u0644\u0627 \u0623\u0646\u0647 \u0628\u0639\u062f \u0633\u0627\u0639\u0627\u062a\u060c \u0627\u0646\u062e\u0641\u0636 \u0627\u0644\u0633\u0639\u0631 \u0625\u0644\u0649 22.52 \u062f\u0648\u0644\u0627\u0631\u064b\u0627 \u0623\u0645\u0631\u064a\u0643\u064a\u064b\u0627 \u0628\u062d\u0644\u0648\u0644 \u0627\u0644\u0633\u0627\u0639\u0629 1:04 \u0645\u0633\u0627\u0621\u064b \u0628\u062a\u0648\u0642\u064a\u062a \u0634\u0631\u0642 \u0627\u0644\u0648\u0644\u0627\u064a\u0627\u062a \u0627\u0644\u0645\u062a\u062d\u062f\u0629. \u0648\u0642\u062f \u0641\u064f\u0633\u0650\u0651\u0631 \u0647\u0630\u0627 \u0627\u0644\u0627\u0646\u062e\u0641\u0627\u0636 \u0639\u0644\u0649 \u0623\u0646\u0647 \u0645\u0624\u0634\u0631 \u0639\u0644\u0649 \u0639\u062f\u0645 \u0631\u0636\u0627 \u0627\u0644\u0645\u0633\u062a\u062b\u0645\u0631\u064a\u0646 \u0639\u0646 \u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0633\u0646\u0648\u0627\u062a \u0627\u0644\u062b\u0644\u0627\u062b \u0644\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Elevidys.<\/p>\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\/creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0625\u0646\u0632\u064a\u0645 \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 \u0643\u064a\u0646\u0627\u0632 (CK) \u0648\u0645\u0633\u062a\u0648\u0649 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0648\u062f\u0631\u062c\u0629 NSAA \u0641\u064a \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Elevidys<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Sarepta announced 3-year topline functional results from patients treated in Part One of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment. Sarepta were not disclosed creatine kinase levels, the most major biomarker [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5485,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[66,33,179,65],"class_list":["post-5484","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duchenne-muscular-dystrophy","tag-elevidys","tag-elevidys-gene-therapy","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/5484","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=5484"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/5484\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/5485"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=5484"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=5484"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=5484"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}