{"id":4399,"date":"2025-09-04T14:17:40","date_gmt":"2025-09-04T11:17:40","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4399"},"modified":"2025-09-04T14:30:44","modified_gmt":"2025-09-04T11:30:44","slug":"sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ru\/sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne\/","title":{"rendered":"SQY Therapeutics \u043f\u043e\u043b\u0443\u0447\u0438\u043b \u0441\u0442\u0430\u0442\u0443\u0441 \u043e\u0440\u0444\u0430\u043d\u043d\u043e\u0433\u043e \u043f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0430 \u043e\u0442 \u0415\u0432\u0440\u043e\u043f\u0435\u0439\u0441\u043a\u043e\u0433\u043e \u0430\u0433\u0435\u043d\u0442\u0441\u0442\u0432\u0430 \u043f\u043e \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u0435\u043d\u043d\u044b\u043c \u0441\u0440\u0435\u0434\u0441\u0442\u0432\u0430\u043c \u0434\u043b\u044f \u0442\u0435\u0440\u0430\u043f\u0438\u0438 \u043f\u0440\u043e\u043f\u0443\u0441\u043a\u0430 \u044d\u043a\u0437\u043e\u043d\u0430 51 SQY51 \u043f\u0440\u0438 \u043b\u0435\u0447\u0435\u043d\u0438\u0438 \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u041f\u0440\u0435\u043f\u0430\u0440\u0430\u0442 SQY51 \u0443\u0436\u0435 \u043f\u043e\u043b\u0443\u0447\u0438\u043b \u0441\u0442\u0430\u0442\u0443\u0441 \u043e\u0440\u0444\u0430\u043d\u043d\u043e\u0433\u043e \u043f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0430 \u043e\u0442 \u0423\u043f\u0440\u0430\u0432\u043b\u0435\u043d\u0438\u044f \u043f\u043e \u043a\u043e\u043d\u0442\u0440\u043e\u043b\u044e \u0437\u0430 \u043f\u0440\u043e\u0434\u0443\u043a\u0442\u0430\u043c\u0438 \u043f\u0438\u0442\u0430\u043d\u0438\u044f \u0438 \u043b\u0435\u043a\u0430\u0440\u0441\u0442\u0432\u0435\u043d\u043d\u044b\u043c\u0438 \u0441\u0440\u0435\u0434\u0441\u0442\u0432\u0430\u043c\u0438 \u0421\u0428\u0410 (FDA) \u0434\u043b\u044f \u043b\u0435\u0447\u0435\u043d\u0438\u044f \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430 \u0432 \u043c\u0430\u0435 2024 \u0433\u043e\u0434\u0430.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SQY51 \u2014 \u044d\u0442\u043e \u043f\u0430\u043b\u044c\u043c\u0438\u0442\u043e\u0438\u043b-\u043a\u043e\u043d\u044a\u044e\u0433\u0438\u0440\u043e\u0432\u0430\u043d\u043d\u044b\u0439 \u0430\u043d\u0442\u0438\u0441\u043c\u044b\u0441\u043b\u043e\u0432\u043e\u0439 \u043e\u043b\u0438\u0433\u043e\u043d\u0443\u043a\u043b\u0435\u043e\u0442\u0438\u0434 \u0442\u0446-\u0414\u041d\u041a, \u043f\u0440\u0435\u0434\u043d\u0430\u0437\u043d\u0430\u0447\u0435\u043d\u043d\u044b\u0439 \u0434\u043b\u044f \u0432\u043e\u0441\u0441\u0442\u0430\u043d\u043e\u0432\u043b\u0435\u043d\u0438\u044f \u043f\u043e\u043b\u0443\u0444\u0443\u043d\u043a\u0446\u0438\u043e\u043d\u0430\u043b\u044c\u043d\u043e\u0433\u043e \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u043d\u0430 \u043f\u0443\u0442\u0435\u043c \u043f\u0440\u043e\u043f\u0443\u0441\u043a\u0430 \u044d\u043a\u0437\u043e\u043d\u0430-51 \u0432\u043e \u0432\u0440\u0435\u043c\u044f \u0441\u043f\u043b\u0430\u0439\u0441\u0438\u043d\u0433\u0430 \u043c\u0420\u041d\u041a \u0433\u0435\u043d\u0430 DMD \u0432 \u0441\u0443\u0431\u043f\u043e\u043f\u0443\u043b\u044f\u0446\u0438\u0438 \u043c\u0430\u043b\u044c\u0447\u0438\u043a\u043e\u0432, \u0441\u0442\u0440\u0430\u0434\u0430\u044e\u0449\u0438\u0445 \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0435\u0439 \u0414\u044e\u0448\u0435\u043d\u043d\u0430.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0427\u0438\u0442\u0430\u0442\u044c \u0434\u0430\u043b\u0435\u0435<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/ru\/exon-51-skipping-therapies-dmd\/\">\u041f\u0440\u0435\u0434\u0441\u0442\u043e\u044f\u0449\u0438\u0435 \u0442\u0435\u0440\u0430\u043f\u0438\u0438 \u043f\u0440\u043e\u043f\u0443\u0441\u043a\u0430 \u044d\u043a\u0437\u043e\u043d\u0430 51 \u0434\u043b\u044f \u043b\u0435\u0447\u0435\u043d\u0438\u044f \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00ab\u041c\u044b \u0440\u0430\u0434\u044b \u043f\u043e\u043b\u0443\u0447\u0438\u0442\u044c \u0441\u0442\u0430\u0442\u0443\u0441 \u043e\u0440\u0444\u0430\u043d\u043d\u043e\u0433\u043e \u043f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0430 \u043e\u0442 EMA, \u0432\u0441\u043b\u0435\u0434 \u0437\u0430 \u0441\u0442\u0430\u0442\u0443\u0441\u043e\u043c, \u043a\u043e\u0442\u043e\u0440\u044b\u0439 \u043c\u044b \u0443\u0436\u0435 \u043f\u043e\u043b\u0443\u0447\u0438\u043b\u0438 \u043e\u0442 FDA\u00bb, \u2014 \u0441\u043a\u0430\u0437\u0430\u043b \u041b\u0443\u0438\u0441 \u0413\u0430\u0440\u0441\u0438\u044f, \u0441\u043f\u0435\u0446\u0438\u0430\u043b\u044c\u043d\u044b\u0439 \u043d\u0430\u0443\u0447\u043d\u044b\u0439 \u043a\u043e\u043d\u0441\u0443\u043b\u044c\u0442\u0430\u043d\u0442 SQY Therapeutics. \u00ab\u042d\u0442\u0438 \u0441\u0442\u0430\u0442\u0443\u0441\u044b \u043e\u0442\u0440\u0430\u0436\u0430\u044e\u0442 \u0437\u043d\u0430\u0447\u0438\u0442\u0435\u043b\u044c\u043d\u0443\u044e \u043d\u0435\u0443\u0434\u043e\u0432\u043b\u0435\u0442\u0432\u043e\u0440\u0435\u043d\u043d\u0443\u044e \u043f\u043e\u0442\u0440\u0435\u0431\u043d\u043e\u0441\u0442\u044c \u0432 \u043f\u0440\u0435\u043f\u0430\u0440\u0430\u0442\u0430\u0445 \u0434\u043b\u044f \u043b\u0435\u0447\u0435\u043d\u0438\u044f \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430 \u0438 \u0443\u043a\u0440\u0435\u043f\u043b\u044f\u044e\u0442 \u043d\u0430\u0448\u0443 \u0432\u0435\u0440\u0443 \u0432 \u043f\u043e\u0442\u0435\u043d\u0446\u0438\u0430\u043b SQY51 \u0434\u043b\u044f \u0441\u0443\u0449\u0435\u0441\u0442\u0432\u0435\u043d\u043d\u043e\u0433\u043e \u0443\u043b\u0443\u0447\u0448\u0435\u043d\u0438\u044f \u0441\u043e\u0441\u0442\u043e\u044f\u043d\u0438\u044f \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u043e\u0432 \u0438 \u0438\u0445 \u0441\u0435\u043c\u0435\u0439, \u0441\u0442\u0440\u0430\u0434\u0430\u044e\u0449\u0438\u0445 \u044d\u0442\u0438\u043c \u0440\u0430\u0437\u0440\u0443\u0448\u0438\u0442\u0435\u043b\u044c\u043d\u044b\u043c \u0437\u0430\u0431\u043e\u043b\u0435\u0432\u0430\u043d\u0438\u0435\u043c \u0438 \u043e\u0431\u0435\u0441\u043f\u043e\u043a\u043e\u0435\u043d\u043d\u044b\u0445 \u043f\u0440\u043e\u043f\u0443\u0441\u043a\u043e\u043c \u044d\u043a\u0437\u043e\u043d\u0430 51\u00bb. <strong>SQY51<\/strong>: <a href=\"https:\/\/clinicaltrials.eu\/inn\/sqy51\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u041f\u0435\u0440\u0441\u043f\u0435\u043a\u0442\u0438\u0432\u043d\u044b\u0439 \u043f\u0440\u0435\u043f\u0430\u0440\u0430\u0442 \u0434\u043b\u044f \u043b\u0435\u0447\u0435\u043d\u0438\u044f \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u041f\u0440\u0435\u043f\u0430\u0440\u0430\u0442 SQY51 \u043f\u0440\u043e\u0445\u043e\u0434\u0438\u0442 \u043e\u0446\u0435\u043d\u043a\u0443 \u0432 \u0440\u0430\u043c\u043a\u0430\u0445 \u043a\u043b\u0438\u043d\u0438\u0447\u0435\u0441\u043a\u043e\u0433\u043e \u0438\u0441\u0441\u043b\u0435\u0434\u043e\u0432\u0430\u043d\u0438\u044f \u0444\u0430\u0437\u044b 1\/2a AVANCE-1 \u0443 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u043e\u0432 \u0441 \u041c\u0414\u0414, \u043f\u043e\u0434\u0434\u0430\u044e\u0449\u0438\u0445\u0441\u044f \u043f\u0440\u043e\u043f\u0443\u0441\u043a\u0443 \u044d\u043a\u0437\u043e\u043d\u0430 51. \u041a\u043e\u043c\u043f\u0430\u043d\u0438\u044f \u0437\u0430\u0432\u0435\u0440\u0448\u0438\u043b\u0430 \u043d\u0430\u0431\u043e\u0440 12 \u043f\u0430\u0446\u0438\u0435\u043d\u0442\u043e\u0432 \u0432 \u0444\u0430\u0437\u0443 1\/2a, \u0434\u0430\u043d\u043d\u044b\u0435 \u043e\u0436\u0438\u0434\u0430\u044e\u0442\u0441\u044f \u0432 2026 \u0433\u043e\u0434\u0443. \u0424\u0430\u0437\u0430 1 \u0437\u0430\u0432\u0435\u0440\u0448\u0435\u043d\u0430, \u0444\u0430\u0437\u0430 2a \u043d\u0430\u0445\u043e\u0434\u0438\u0442\u0441\u044f \u0432 \u043f\u0440\u043e\u0446\u0435\u0441\u0441\u0435 \u0440\u0435\u0430\u043b\u0438\u0437\u0430\u0446\u0438\u0438. SQY Therapeutics \u0442\u0430\u043a\u0436\u0435 \u043f\u043b\u0430\u043d\u0438\u0440\u0443\u0435\u0442 \u043f\u0440\u043e\u0434\u043b\u0435\u043d\u0438\u0435 \u0444\u0430\u0437\u044b \u0434\u043b\u044f \u0443\u0447\u0430\u0441\u0442\u043d\u0438\u043a\u043e\u0432, \u0436\u0435\u043b\u0430\u044e\u0449\u0438\u0445 \u043f\u0440\u043e\u0434\u043e\u043b\u0436\u0438\u0442\u044c \u043b\u0435\u0447\u0435\u043d\u0438\u0435.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0423\u0437\u043d\u0430\u0442\u044c \u0431\u043e\u043b\u044c\u0448\u0435<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/ru\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">\u0420\u0430\u0437\u0440\u0430\u0431\u043e\u0442\u0430\u043d\u044b \u043c\u0435\u0442\u043e\u0434\u044b \u043f\u0440\u043e\u043f\u0443\u0441\u043a\u0430 \u044d\u043a\u0437\u043e\u043d\u043e\u0432 \u043d\u043e\u0432\u043e\u0433\u043e \u043f\u043e\u043a\u043e\u043b\u0435\u043d\u0438\u044f \u0434\u043b\u044f \u043b\u0435\u0447\u0435\u043d\u0438\u044f \u043c\u044b\u0448\u0435\u0447\u043d\u043e\u0439 \u0434\u0438\u0441\u0442\u0440\u043e\u0444\u0438\u0438 \u0414\u044e\u0448\u0435\u043d\u043d\u0430<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>SQY51 had already obtained Orphan Drug Designation from the US Food and Drug Administration (FDA) in Duchenne Muscular Dystrophy in May 2024. SQY51 is a palmitoyl-conjugated tc-DNA antisense oligonucleotide designed to restore semi-functional dystrophin by skipping exon-51 during mRNA splicing of the DMD gene in a subpopulation of boys affected by Duchenne Muscular Dystrophy. Read [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4403,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[91,213,122,193,194,410,195,409,408],"class_list":["post-4399","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ema","tag-european-medicines-agency","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-france","tag-orphan-drug","tag-sqy-therapeutics","tag-sqy51"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts\/4399","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/comments?post=4399"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/posts\/4399\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/media\/4403"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/media?parent=4399"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/categories?post=4399"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ru\/wp-json\/wp\/v2\/tags?post=4399"}],"curies":[{"name":"WP","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}