{"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\/hi\/sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne\/","title":{"rendered":"SQY Therapeutics \u0915\u094b \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 SQY51 \u0915\u0947 \u0932\u093f\u090f \u092f\u0942\u0930\u094b\u092a\u0940\u092f \u092e\u0947\u0921\u093f\u0938\u093f\u0928 \u090f\u091c\u0947\u0902\u0938\u0940 \u0938\u0947 \u0905\u0928\u093e\u0925 \u0926\u0935\u093e \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u0941\u0906 \u0939\u0948\u0964"},"content":{"rendered":"

\u090f\u0938\u0915\u094d\u092f\u0942\u0935\u093e\u090851 \u0915\u094b \u092e\u0908 2024 \u092e\u0947\u0902 \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u092e\u0947\u0902 \u0905\u092e\u0947\u0930\u093f\u0915\u0940 \u0916\u093e\u0926\u094d\u092f \u090f\u0935\u0902 \u0914\u0937\u0927\u093f \u092a\u094d\u0930\u0936\u093e\u0938\u0928 (1\u091f\u0940\u092a\u0940121\u091f\u0940) \u0938\u0947 \u0905\u0928\u093e\u0925 \u0926\u0935\u093e \u092a\u0926\u0928\u093e\u092e \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u094b \u091a\u0941\u0915\u093e \u0939\u0948\u0964<\/p>\n\n\n\n

\u090f\u0938\u0915\u094d\u092f\u0942\u0935\u093e\u090851 \u090f\u0915 \u092a\u093e\u092e\u093f\u091f\u094b\u092f\u0932-\u0938\u0902\u092f\u0941\u0917\u094d\u092e\u093f\u0924 \u091f\u0940\u0938\u0940-\u0921\u0940\u090f\u0928\u090f \u090f\u0902\u091f\u0940\u0938\u0947\u0928\u094d\u0938 \u0911\u0932\u093f\u0917\u094b\u0928\u094d\u092f\u0941\u0915\u094d\u0932\u093f\u092f\u094b\u091f\u093e\u0907\u0921 \u0939\u0948, \u091c\u093f\u0938\u0947 \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0938\u0947 \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0932\u0921\u093c\u0915\u094b\u0902 \u0915\u0940 \u0909\u092a-\u091c\u0928\u0938\u0902\u0916\u094d\u092f\u093e \u092e\u0947\u0902 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u0915\u0947 \u090f\u092e\u0906\u0930\u090f\u0928\u090f \u0938\u094d\u092a\u094d\u0932\u093f\u0938\u093f\u0902\u0917 \u0915\u0947 \u0926\u094c\u0930\u093e\u0928 \u090f\u0915\u094d\u0938\u0949\u0928-51 \u0915\u094b \u091b\u094b\u0921\u093c\u0915\u0930 \u0905\u0930\u094d\u0927-\u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948\u0964<\/p>\n\n\n\n

\u0914\u0930 \u092a\u0922\u093c\u0947\u0902<\/strong>: \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0906\u0917\u093e\u092e\u0940 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940<\/a><\/p>\n\n\n\n

SQY Therapeutics \u0915\u0947 \u0935\u093f\u0936\u0947\u0937 \u0935\u0948\u091c\u094d\u091e\u093e\u0928\u093f\u0915 \u0938\u0932\u093e\u0939\u0915\u093e\u0930 \u0932\u0941\u0907\u0938 \u0917\u093e\u0930\u094d\u0938\u093f\u092f\u093e \u0928\u0947 \u0915\u0939\u093e, "\u0939\u092e\u0947\u0902 EMA \u0938\u0947 \u0911\u0930\u094d\u092b\u0928 \u0921\u094d\u0930\u0917 \u0921\u093f\u091c\u093c\u093f\u0917\u0947\u0936\u0928 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930\u0915\u0947 \u0916\u0941\u0936\u0940 \u0939\u094b \u0930\u0939\u0940 \u0939\u0948, \u091c\u094b FDA \u0938\u0947 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0921\u093f\u091c\u093c\u093f\u0917\u0947\u0936\u0928 \u0915\u0947 \u092c\u093e\u0926 \u0906\u092f\u093e \u0939\u0948\u0964" \u0909\u0928\u094d\u0939\u094b\u0902\u0928\u0947 \u0906\u0917\u0947 \u0915\u0939\u093e, "\u092f\u0947 \u0921\u093f\u091c\u093c\u093f\u0917\u0947\u0936\u0928 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0940 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0905\u092a\u0942\u0930\u094d\u0923 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0915\u094b \u0926\u0930\u094d\u0936\u093e\u0924\u0947 \u0939\u0948\u0902 \u0914\u0930 \u0907\u0938 \u0935\u093f\u0928\u093e\u0936\u0915\u093e\u0930\u0940 \u092c\u0940\u092e\u093e\u0930\u0940 \u0938\u0947 \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0914\u0930 \u090f\u0915\u094d\u0938\u0949\u0928-51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0938\u0947 \u091a\u093f\u0902\u0924\u093f\u0924 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0914\u0930 \u092a\u0930\u093f\u0935\u093e\u0930\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0938\u093e\u0930\u094d\u0925\u0915 \u092c\u0926\u0932\u093e\u0935 \u0932\u093e\u0928\u0947 \u0915\u0940 SQY51 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u092e\u0947\u0902 \u0939\u092e\u093e\u0930\u0947 \u0935\u093f\u0936\u094d\u0935\u093e\u0938 \u0915\u094b \u092a\u0941\u0937\u094d\u091f \u0915\u0930\u0924\u0947 \u0939\u0948\u0902\u0964" \u090f\u0938\u0915\u094d\u092f\u0942\u0935\u093e\u090851<\/strong>: \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u0906\u0936\u093e\u091c\u0928\u0915 \u0926\u0935\u093e<\/a><\/p>\n\n\n\n

\u090f\u0938\u0915\u094d\u092f\u0942\u0935\u093e\u090851 \u0915\u093e \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u0921\u0940\u090f\u092e\u0921\u0940 \u0938\u0947 \u092a\u0940\u0921\u093c\u093f\u0924 \u0909\u0928 \u0935\u094d\u092f\u0915\u094d\u0924\u093f\u092f\u094b\u0902 \u092a\u0930 \u091a\u0930\u0923 1\/2\u090f \u090f\u0935\u0947\u0902\u0938-1 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u092e\u0947\u0902 \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948 \u091c\u094b \u090f\u0915\u094d\u0938\u0949\u0928-51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947 \u0932\u093f\u090f \u0909\u0924\u094d\u0924\u0930\u0926\u093e\u092f\u0940 \u0939\u0948\u0902\u0964 \u0915\u0902\u092a\u0928\u0940 \u0928\u0947 \u091a\u0930\u0923 1\/2\u090f \u092e\u0947\u0902 12 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u093e \u0928\u093e\u092e\u093e\u0902\u0915\u0928 \u092a\u0942\u0930\u093e \u0915\u0930 \u0932\u093f\u092f\u093e \u0939\u0948, \u0914\u0930 \u0921\u0947\u091f\u093e 2026 \u092e\u0947\u0902 \u0906\u0928\u0947 \u0915\u0940 \u0909\u092e\u094d\u092e\u0940\u0926 \u0939\u0948\u0964 \u091a\u0930\u0923 1 \u092a\u0942\u0930\u093e \u0939\u094b \u091a\u0941\u0915\u093e \u0939\u0948 \u0914\u0930 \u091a\u0930\u0923 2\u090f \u091a\u0932 \u0930\u0939\u093e \u0939\u0948\u0964 1\u091f\u0940\u092a\u094039\u091f\u0940 \u0909\u0928 \u092a\u094d\u0930\u0924\u093f\u092d\u093e\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u0935\u093f\u0938\u094d\u0924\u093e\u0930 \u091a\u0930\u0923 \u0915\u0940 \u092d\u0940 \u092f\u094b\u091c\u0928\u093e \u092c\u0928\u093e \u0930\u0939\u093e \u0939\u0948 \u091c\u094b \u0909\u092a\u091a\u093e\u0930 \u0938\u0947 \u0932\u093e\u092d \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0915\u0930\u0928\u093e \u091c\u093e\u0930\u0940 \u0930\u0916\u0928\u093e \u091a\u093e\u0939\u0924\u0947 \u0939\u0948\u0902\u0964<\/p>\n\n\n\n

\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902<\/strong>: \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0940 \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0935\u093f\u0915\u0938\u093f\u0924 \u0915\u0940 \u0917\u0908<\/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":"SQY Therapeutics \u0928\u0947 \u0918\u094b\u0937\u0923\u093e \u0915\u0940 \u0915\u093f \u092f\u0942\u0930\u094b\u092a\u0940\u092f \u092e\u0947\u0921\u093f\u0938\u093f\u0928 \u090f\u091c\u0947\u0902\u0938\u0940 (EMA) \u0928\u0947 SQY51 \u0915\u094b \u0911\u0930\u094d\u092b\u0928 \u0921\u094d\u0930\u0917 \u0921\u0947\u091c\u093f\u0917\u094d\u0928\u0947\u0936\u0928 (ODD) \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u093f\u092f\u093e \u0939\u0948, \u091c\u094b \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0915\u0947 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u0940-\u090f\u092e\u0906\u0930\u090f\u0928\u090f \u0915\u0947 \u090f\u0915\u094d\u0938\u0949\u0928-51 \u0915\u094b \u0932\u0915\u094d\u0937\u093f\u0924 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u093e \u090f\u0915 \u0905\u0902\u0924\u0903\u0936\u093f\u0930\u093e \u0905\u0923\u0941 \u0939\u0948\u0964","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/4399","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/comments?post=4399"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/4399\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/4403"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=4399"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=4399"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=4399"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}