{"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\/ar\/sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne\/","title":{"rendered":"\u062d\u0635\u0644 SQY Therapeutics \u0639\u0644\u0649 \u062a\u0635\u0646\u064a\u0641 \u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645 \u0645\u0646 \u0648\u0643\u0627\u0644\u0629 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629 \u0644\u0639\u0644\u0627\u062c \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51 SQY51 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646"},"content":{"rendered":"

\u062d\u0635\u0644 SQY51 \u0628\u0627\u0644\u0641\u0639\u0644 \u0639\u0644\u0649 \u062a\u0635\u0646\u064a\u0641 \u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u064a\u062a\u064a\u0645 \u0645\u0646 \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 (FDA) \u0641\u064a \u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 \u0641\u064a \u0645\u0627\u064a\u0648 2024.<\/p>\n\n\n\n

SQY51 \u0647\u0648 \u0623\u0648\u0644\u064a\u062c\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f \u0645\u0636\u0627\u062f \u0644\u0644\u0645\u0639\u0646\u0649 \u0645\u0646 \u0646\u0648\u0639 TC-DNA \u0645\u0642\u062a\u0631\u0646 \u0628\u0627\u0644\u0628\u0627\u0644\u0645\u064a\u062a\u0648\u064a\u0644 \u0645\u0635\u0645\u0645 \u0644\u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0634\u0628\u0647 \u0627\u0644\u0648\u0638\u064a\u0641\u064a \u0639\u0646 \u0637\u0631\u064a\u0642 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0623\u062b\u0646\u0627\u0621 \u0631\u0628\u0637 mRNA \u0644\u062c\u064a\u0646 DMD \u0641\u064a \u0645\u062c\u0645\u0648\u0639\u0629 \u0641\u0631\u0639\u064a\u0629 \u0645\u0646 \u0627\u0644\u0623\u0648\u0644\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.<\/p>\n\n\n\n

\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f<\/strong>: \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51 \u0627\u0644\u0642\u0627\u062f\u0645\u0629 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>\n\n\n\n

\u0642\u0627\u0644 \u0644\u0648\u064a\u0633 \u063a\u0627\u0631\u0633\u064a\u0627\u060c \u0627\u0644\u0645\u0633\u062a\u0634\u0627\u0631 \u0627\u0644\u0639\u0644\u0645\u064a \u0627\u0644\u062e\u0627\u0635 \u0644\u0640 SQY Therapeutics: "\u064a\u0633\u0639\u062f\u0646\u0627 \u0627\u0644\u062d\u0635\u0648\u0644 \u0639\u0644\u0649 \u062a\u0635\u0646\u064a\u0641 \u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645 \u0645\u0646 EMA\u060c \u0648\u0630\u0644\u0643 \u0628\u0639\u062f \u0627\u0644\u062a\u0635\u0646\u064a\u0641 \u0627\u0644\u0630\u064a \u062d\u0635\u0644\u0646\u0627 \u0639\u0644\u064a\u0647 \u0633\u0627\u0628\u0642\u064b\u0627 \u0645\u0646 FDA". \u0648\u0623\u0636\u0627\u0641: "\u062a\u0639\u0643\u0633 \u0647\u0630\u0647 \u0627\u0644\u062a\u0635\u0646\u064a\u0641\u0627\u062a \u0627\u0644\u062d\u0627\u062c\u0629 \u0627\u0644\u0645\u0644\u062d\u0629 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646\u060c \u0648\u062a\u0639\u0632\u0632 \u0625\u064a\u0645\u0627\u0646\u0646\u0627 \u0628\u0625\u0645\u0643\u0627\u0646\u064a\u0629 \u0625\u062d\u062f\u0627\u062b SQY51 \u0641\u0631\u0642\u064b\u0627 \u0645\u0644\u0645\u0648\u0633\u064b\u0627 \u0644\u0644\u0645\u0631\u0636\u0649 \u0648\u0639\u0627\u0626\u0644\u0627\u062a\u0647\u0645 \u0627\u0644\u0645\u062a\u0623\u062b\u0631\u064a\u0646 \u0628\u0647\u0630\u0627 \u0627\u0644\u0645\u0631\u0636 \u0627\u0644\u062e\u0637\u064a\u0631\u060c \u0648\u0627\u0644\u0630\u064a\u0646 \u064a\u0634\u0639\u0631\u0648\u0646 \u0628\u0627\u0644\u0642\u0644\u0642 \u0625\u0632\u0627\u0621 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646-51". SQY51<\/strong>: \u062f\u0648\u0627\u0621 \u0648\u0627\u0639\u062f \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>\n\n\n\n

\u064a\u062c\u0631\u064a \u062a\u0642\u064a\u064a\u0645 SQY51 \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2\u0623 \u0645\u0646 \u062a\u062c\u0631\u0628\u0629 AVANCE-1 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0639\u0644\u0649 \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0627\u0644\u0630\u064a\u0646 \u064a\u0645\u0643\u0646\u0647\u0645 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646-51. \u0648\u0642\u062f \u0623\u0643\u0645\u0644\u062a \u0627\u0644\u0634\u0631\u0643\u0629 \u062a\u0633\u062c\u064a\u0644 12 \u0645\u0631\u064a\u0636\u064b\u0627 \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2\u0623\u060c \u0648\u0645\u0646 \u0627\u0644\u0645\u062a\u0648\u0642\u0639 \u0635\u062f\u0648\u0631 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0641\u064a \u0639\u0627\u0645 2026. \u0648\u0642\u062f \u0627\u0643\u062a\u0645\u0644\u062a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\u060c \u0648\u0627\u0644\u0645\u0631\u062d\u0644\u0629 2\u0623 \u0642\u064a\u062f \u0627\u0644\u062a\u0646\u0641\u064a\u0630. \u0643\u0645\u0627 \u062a\u062e\u0637\u0637 SQY Therapeutics \u0644\u0645\u0631\u062d\u0644\u0629 \u062a\u0645\u062f\u064a\u062f \u0644\u0644\u0645\u0634\u0627\u0631\u0643\u064a\u0646 \u0627\u0644\u0630\u064a\u0646 \u064a\u0631\u063a\u0628\u0648\u0646 \u0641\u064a \u0645\u0648\u0627\u0635\u0644\u0629 \u0627\u0644\u0627\u0633\u062a\u0641\u0627\u062f\u0629 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c.<\/p>\n\n\n\n

\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631<\/strong>: \u062a\u0645 \u062a\u0637\u0648\u064a\u0631 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u0642\u0627\u062f\u0645 \u0645\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>","protected":false},"excerpt":{"rendered":"

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 […]<\/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"],"subtitle":"\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 SQY Therapeutics \u0623\u0646 \u0648\u0643\u0627\u0644\u0629 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629 (EMA) \u0645\u0646\u062d\u062a \u062a\u0635\u0646\u064a\u0641 \u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u064a\u062a\u064a\u0645 (ODD) \u0625\u0644\u0649 SQY51\u060c \u0648\u0647\u0648 \u062c\u0632\u064a\u0621 \u0648\u0631\u064a\u062f\u064a \u064a\u0633\u062a\u0647\u062f\u0641 \u0625\u0643\u0633\u0648\u0646 51 \u0645\u0646 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u0648\u0632\u064a \u0627\u0644\u0645\u0631\u0633\u0627\u0644 \u0644\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/4399","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=4399"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/4399\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/4403"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=4399"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=4399"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=4399"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}