{"id":3429,"date":"2025-03-31T12:19:41","date_gmt":"2025-03-31T09:19:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3429"},"modified":"2025-11-03T20:22:47","modified_gmt":"2025-11-03T17:22:47","slug":"exon-51-skipping-therapies-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/hi\/exon-51-skipping-therapies-dmd\/","title":{"rendered":"\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"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0905\u092d\u093f\u0928\u0935 \u0909\u092a\u091a\u093e\u0930 \u0939\u0948\u0902 \u091c\u093f\u0928\u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (\u0921\u0940\u090f\u092e\u0921\u0940) \u0915\u0947 \u0932\u093f\u090f \u091c\u093f\u092e\u094d\u092e\u0947\u0926\u093e\u0930 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0915\u094b \u0938\u0902\u092c\u094b\u0927\u093f\u0924 \u0915\u0930\u0928\u093e \u0939\u0948\u0964 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u0938\u094d\u0915\u093f\u092a \u0915\u0930\u0915\u0947, \u092f\u0947 \u0925\u0947\u0930\u0947\u092a\u0940 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u090f\u0915 \u091b\u094b\u091f\u093e, \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0902\u0938\u094d\u0915\u0930\u0923 \u092c\u0928\u093e\u0928\u0947 \u092e\u0947\u0902 \u092e\u0926\u0926 \u0915\u0930\u0924\u0940 \u0939\u0948, \u091c\u094b \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u0930\u094b\u0917 \u0915\u0940 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u094b \u0927\u0940\u092e\u093e \u0915\u0930 \u0926\u0947\u0924\u0940 \u0939\u0948 \u0914\u0930 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u091c\u0940\u0935\u0928 \u0915\u0940 \u0917\u0941\u0923\u0935\u0924\u094d\u0924\u093e \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0915\u0930\u0924\u0940 \u0939\u0948\u0964 \u092f\u0939 \u0905\u0924\u094d\u092f\u093e\u0927\u0941\u0928\u093f\u0915 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0921\u0940\u090f\u092e\u0921\u0940 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0906\u0936\u093e \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0924\u093e \u0939\u0948, \u091c\u094b \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0947 \u0915\u093e\u0930\u094d\u092f \u0915\u094b \u092c\u0922\u093c\u093e\u0928\u0947 \u0914\u0930 \u0917\u0924\u093f\u0936\u0940\u0932\u0924\u093e \u0915\u094b \u0932\u092e\u094d\u092c\u093e \u0915\u0930\u0928\u0947 \u092a\u0930 \u0927\u094d\u092f\u093e\u0928 \u0915\u0947\u0902\u0926\u094d\u0930\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0935\u093f\u0937\u092f\u0938\u0942\u091a\u0940<\/h2><nav><ul><li><a href=\"#about-duchenne\">\u0921\u091a\u0947\u0928 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902<\/a><\/li><li><a href=\"#exon-51-skipping-therapies\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940<\/a><\/li><li><a href=\"#what-is-exon-51-skipping\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u094d\u092f\u093e \u0939\u0948?<\/a><\/li><li><a href=\"#fda-approved-exon-51-skipping-therapies\">FDA-\u0905\u0928\u0941\u092e\u094b\u0926\u093f\u0924 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940<\/a><ul><li><a href=\"#exondys-51-eteplirsen\">EXONDYS 51 (eteplirsen)<\/a><\/li><\/ul><\/li><li><a href=\"#which-mutations-are-amenable-to-exon-45-skipping\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u091b\u094b\u0921\u093c\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u0935\u093f\u0932\u094b\u092a\u0928<\/a><\/li><li><a href=\"#clinical-trials-for-exon-51-skipping-therapies\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u092a\u0930\u0940\u0915\u094d\u0937\u0923<\/a><ul><li><a href=\"#dyne-therapeutics-dyne-251-exon-51\">1\u091f\u0940\u092a\u094016\u091f\u0940 \u2013 1\u091f\u0940\u092a\u094049\u091f\u0940<\/a><\/li><li><a href=\"#jiao-tong-university-le-051\">\u091c\u093f\u0913 \u091f\u094b\u0902\u0917 \u0935\u093f\u0936\u094d\u0935\u0935\u093f\u0926\u094d\u092f\u093e\u0932\u092f - LE051<\/a><\/li><li><a href=\"#bio-marin-pharmaceutical-bmn-351\">\u092c\u093e\u092f\u094b\u092e\u0930\u0940\u0928 \u092b\u093e\u0930\u094d\u092e\u093e\u0938\u094d\u092f\u0941\u091f\u093f\u0915\u0932 \u2013 BMN 351<\/a><\/li><li><a href=\"#entrada-therapeutics\">Entrada Therapeutics \u2013 ENTR-601-51<\/a><\/li><li><a href=\"#nippon-shinyaku-ns-051-ncnp-04\">Nippon Shinyaku \u2013 \u090f\u0928\u090f\u0938-051\/\u090f\u0928\u0938\u0940\u090f\u0928\u092a\u0940-04<\/a><\/li><\/ul><\/li><li><a href=\"#conclusion\">\u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"about-duchenne\">\u0921\u091a\u0947\u0928 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (\u0921\u0940\u090f\u092e\u0921\u0940) \u090f\u0915 \u0917\u0902\u092d\u0940\u0930 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0935\u093f\u0915\u093e\u0930 \u0939\u0948 \u091c\u094b \u092e\u0941\u0916\u094d\u092f \u0930\u0942\u092a \u0938\u0947 \u0932\u0921\u093c\u0915\u094b\u0902 \u0915\u094b \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948 \u0914\u0930 \u092a\u094d\u0930\u0917\u0924\u093f\u0936\u0940\u0932 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0915\u092e\u091c\u094b\u0930\u0940, \u0917\u0924\u093f\u0936\u0940\u0932\u0924\u093e \u0915\u0940 \u0939\u093e\u0928\u093f \u0914\u0930 \u0905\u0902\u0924\u0924\u0903 \u0936\u094d\u0935\u0938\u0928 \u0914\u0930 \u0939\u0943\u0926\u092f \u0938\u0902\u092c\u0902\u0927\u0940 \u091c\u091f\u093f\u0932\u0924\u093e\u0913\u0902 \u0915\u093e \u0915\u093e\u0930\u0923 \u092c\u0928\u0924\u093e \u0939\u0948\u0964 \u0921\u0940\u090f\u092e\u0921\u0940 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u092e\u0947\u0902 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0915\u0947 \u0915\u093e\u0930\u0923 \u0939\u094b\u0924\u093e \u0939\u0948, \u091c\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u094b \u090f\u0928\u0915\u094b\u0921 \u0915\u0930\u0924\u093e \u0939\u0948, \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u091c\u094b \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0915\u094b\u0936\u093f\u0915\u093e \u091d\u093f\u0932\u094d\u0932\u0940 \u0915\u094b \u0938\u094d\u0925\u093f\u0930 \u0915\u0930\u0924\u093e \u0939\u0948\u0964 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0947 \u092c\u093f\u0928\u093e, \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u092b\u093e\u0907\u092c\u0930 \u0938\u0902\u0915\u0941\u091a\u0928 \u0915\u0947 \u0926\u094c\u0930\u093e\u0928 \u0915\u094d\u0937\u0924\u093f \u0915\u0947 \u0932\u093f\u090f \u0905\u0927\u093f\u0915 \u092a\u094d\u0930\u0935\u0923 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902, \u091c\u093f\u0938\u0938\u0947 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0905\u0927:\u092a\u0924\u0928 \u0914\u0930 \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u092a\u094d\u0930\u092e\u0941\u0916 \u0932\u0915\u094d\u0937\u0923 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902\u0964<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"exon-51-skipping-therapies\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0921\u0940\u090f\u092e\u0921\u0940 \u092e\u0947\u0902 \u092e\u0941\u0916\u094d\u092f \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u092e\u0947\u0902 \u0935\u093f\u0936\u093f\u0937\u094d\u091f \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948, \u091c\u094b \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0947 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u094b \u092c\u093e\u0927\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948\u0964 \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0939\u0948, \u091c\u094b \u090f\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u0930\u0923\u0928\u0940\u0924\u093f \u0939\u0948 \u091c\u093f\u0938\u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u091c\u0940\u0928 \u0915\u0947 \u0926\u094b\u0937\u092a\u0942\u0930\u094d\u0923 \u092d\u093e\u0917\u094b\u0902 \u0915\u094b \u092c\u093e\u092f\u092a\u093e\u0938 \u0915\u0930\u0928\u093e \u0939\u0948 \u0924\u093e\u0915\u093f \u090f\u0915 \u091b\u094b\u091f\u093e, \u0932\u0947\u0915\u093f\u0928 \u0906\u0902\u0936\u093f\u0915 \u0930\u0942\u092a \u0938\u0947 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915, \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0939\u094b \u0938\u0915\u0947\u0964 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0938\u092c\u0938\u0947 \u0905\u0927\u093f\u0915 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u0940 \u0917\u0908 \u090f\u0915\u094d\u0938\u0949\u0928-\u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u092e\u0947\u0902 \u0938\u0947 \u090f\u0915 \u0939\u0948, \u091c\u094b \u0921\u0940\u090f\u092e\u0921\u0940 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0909\u092a\u0938\u092e\u0942\u0939 \u0915\u094b \u0906\u0936\u093e \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0924\u0940 \u0939\u0948\u0964 (<a href=\"https:\/\/dmdwarrior.com\/hi\/what-is-exon-skipping-and-how-does-it-work\/\" data-type=\"post\" data-id=\"1445\">\u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u094d\u092f\u093e \u0939\u0948?<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-exon-51-skipping\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u094d\u092f\u093e \u0939\u0948?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u092e\u0947\u0902 79 \u090f\u0915\u094d\u0938\u0949\u0928 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902, \u0914\u0930 \u0907\u0938 \u091c\u0940\u0928 \u092e\u0947\u0902 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u092e\u0947\u0902 \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u0935\u093f\u0936\u093f\u0937\u094d\u091f \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u093e \u0935\u093f\u0932\u094b\u092a\u0928 \u0936\u093e\u092e\u093f\u0932 \u0939\u094b\u0924\u093e \u0939\u0948\u0964 \u0907\u0928 \u0935\u093f\u0932\u094b\u092a\u0928\u094b\u0902 \u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e\u0938\u094d\u0935\u0930\u0942\u092a \u0906\u092e\u0924\u094c\u0930 \u092a\u0930 \u090f\u0915 \u092b\u093c\u094d\u0930\u0947\u092e\u0936\u093f\u092b\u094d\u091f \u0939\u094b\u0924\u093e \u0939\u0948 \u091c\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u0915\u0947 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u094b \u0930\u094b\u0915\u0924\u093e \u0939\u0948\u0964 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u092e\u0947\u0902 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u093f\u0924 \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u094b \u091b\u093f\u092a\u093e\u0928\u0947 \u0914\u0930 \u0906\u0930\u090f\u0928\u090f \u0915\u0940 \u0938\u094d\u092a\u094d\u0932\u093f\u0938\u093f\u0902\u0917 \u092a\u094d\u0930\u0915\u094d\u0930\u093f\u092f\u093e \u0915\u0947 \u0926\u094c\u0930\u093e\u0928 \u0909\u0938 \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u0947 &quot;\u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917&quot; \u0915\u094b \u092c\u0922\u093c\u093e\u0935\u093e \u0926\u0947\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u090f\u0902\u091f\u0940\u0938\u0947\u0902\u0938 \u0911\u0932\u093f\u0917\u094b\u0928\u094d\u092f\u0942\u0915\u094d\u0932\u093f\u092f\u094b\u091f\u093e\u0907\u0921\u094d\u0938 (\u090f\u090f\u0938\u0913) \u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u0930\u0928\u093e \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0964 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u0938\u094d\u0915\u093f\u092a \u0915\u0930\u0915\u0947, \u0921\u093e\u0909\u0928\u0938\u094d\u091f\u094d\u0930\u0940\u092e \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u094b \u090f\u0915 \u0938\u093e\u0925 \u091c\u094b\u0921\u093c\u093e \u091c\u093e \u0938\u0915\u0924\u093e \u0939\u0948, \u091c\u093f\u0938\u0938\u0947 \u0930\u0940\u0921\u093f\u0902\u0917 \u092b\u093c\u094d\u0930\u0947\u092e \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u093f\u092f\u093e \u091c\u093e \u0938\u0915\u0924\u093e \u0939\u0948 \u0914\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u0915\u0947 \u090f\u0915 \u091b\u094b\u091f\u0947 \u0932\u0947\u0915\u093f\u0928 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0930\u0942\u092a \u0915\u0947 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u0940 \u0905\u0928\u0941\u092e\u0924\u093f \u092e\u093f\u0932\u0924\u0940 \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0907\u0938 \u0930\u0923\u0928\u0940\u0924\u093f \u0915\u093e \u0909\u0926\u094d\u0926\u0947\u0936\u094d\u092f \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u0930\u0928\u093e \u0939\u0948 \u091c\u094b \u0938\u093e\u092e\u093e\u0928\u094d\u092f \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0940 \u0915\u092e\u0940 \u0915\u0940 \u0915\u0941\u091b \u0939\u0926 \u0924\u0915 \u092d\u0930\u092a\u093e\u0908 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948, \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u0930\u094b\u0917 \u0915\u0940 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u094b \u0915\u092e \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948 \u0914\u0930 \u0909\u0928 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0947 \u0915\u093e\u0930\u094d\u092f \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948 \u091c\u093f\u0928\u0915\u0947 \u0935\u093f\u0932\u094b\u092a\u0928 \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 (\u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/what-is-exon-deletion-in-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0921\u0940\u090f\u092e\u0921\u0940 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 \u0935\u093f\u0932\u094b\u092a\u0928 \u0915\u094d\u092f\u093e \u0939\u0948?<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"fda-approved-exon-51-skipping-therapies\">FDA-\u0905\u0928\u0941\u092e\u094b\u0926\u093f\u0924 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"exondys-51-eteplirsen\">EXONDYS 51 (eteplirsen)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">EXONDYS 51 (\u090f\u091f\u0947\u092a\u094d\u0932\u093f\u0930\u094d\u0938\u0928) \u0909\u0928 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u092a\u0939\u0932\u093e FDA-\u0938\u094d\u0935\u0940\u0915\u0943\u0924 \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u0909\u092a\u091a\u093e\u0930 \u0939\u0948, \u091c\u093f\u0928\u0915\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915 \u092a\u0941\u0937\u094d\u091f\u093f\u0915\u0943\u0924 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0939\u0948, \u091c\u093f\u0938\u0915\u093e \u0907\u0932\u093e\u091c \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u091b\u094b\u0921\u093c\u0915\u0930 \u0915\u093f\u092f\u093e \u091c\u093e \u0938\u0915\u0924\u093e \u0939\u0948\u0964 \u0915\u0941\u091b \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902, \u092f\u0939 \u0936\u0930\u0940\u0930 \u0915\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u090f\u0915 \u091b\u094b\u091f\u093e \u0930\u0942\u092a \u092c\u0928\u093e\u0928\u0947 \u092e\u0947\u0902 \u092e\u0926\u0926 \u0915\u0930\u0924\u093e \u0939\u0948\u0964 (\u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/frequently-asked-questions-about-eteplirsen-exondys-51-for-dmd-treatment\/\" data-type=\"post\" data-id=\"2303\">Eteplirsen<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u090f\u0915\u094d\u0938\u094b\u0928\u094d\u0921\u093f\u0938 51 (\u090f\u091f\u0947\u092a\u094d\u0932\u093f\u0930\u094d\u0938\u0947\u0928) \u0915\u094b \u092f\u0942\u0930\u094b\u092a\u0940\u092f \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u090f\u091c\u0947\u0902\u0938\u0940 \u0926\u094d\u0935\u093e\u0930\u093e \u0905\u0928\u0941\u092e\u094b\u0926\u093f\u0924 \u0928\u0939\u0940\u0902 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0925\u093e\u0964<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/exondys\" target=\"_blank\" rel=\"noopener\">1\u091f\u0940\u092a\u094010\u091f\u0940<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"which-mutations-are-amenable-to-exon-45-skipping\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u091b\u094b\u0921\u093c\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u0935\u093f\u0932\u094b\u092a\u0928<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0935\u093f\u0936\u0947\u0937 \u0930\u0942\u092a \u0938\u0947 \u0909\u0928 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0921\u093f\u091c\u093c\u093e\u0907\u0928 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948 \u091c\u093f\u0928\u0915\u0947 DMD \u091c\u0940\u0928 \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 \u092e\u0947\u0902 \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948, \u091c\u093f\u0938\u0947 \u0906\u092a \u0928\u0940\u091a\u0947 \u0926\u0947\u0916 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902\u0964 \u092f\u0947 \u0935\u093f\u0932\u094b\u092a\u0928 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u0939\u0948\u0902, \u0915\u094d\u092f\u094b\u0902\u0915\u093f \u0907\u0938 \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u094b \u091b\u094b\u0921\u093c\u0928\u0947 \u0938\u0947 \u091c\u0940\u0928 \u0915\u093e \u0930\u0940\u0921\u093f\u0902\u0917 \u092b\u094d\u0930\u0947\u092e \u092c\u0939\u093e\u0932 \u0939\u094b \u091c\u093e\u0924\u093e \u0939\u0948, \u091c\u093f\u0938\u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e\u0938\u094d\u0935\u0930\u0942\u092a \u090f\u0915 \u091b\u094b\u091f\u093e, \u0932\u0947\u0915\u093f\u0928 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915, \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u093e \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0939\u094b\u0924\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img decoding=\"async\" width=\"847\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg\" alt=\"\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\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0905\u0928\u0941\u0915\u0942\u0932 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0914\u0930 \u0935\u093f\u0932\u094b\u092a\u0928\" class=\"wp-image-4526\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg 847w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-248x300.jpg 248w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-768x929.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1270x1536.jpg 1270w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1693x2048.jpg 1693w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-10x12.jpg 10w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-347x420.jpg 347w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-150x181.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-300x363.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-696x842.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1068x1292.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1920x2323.jpg 1920w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping.jpg 1984w\" sizes=\"(max-width: 847px) 100vw, 847px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">\u0936\u094b\u0927 \u0938\u0947 \u092a\u0924\u093e \u091a\u0932\u093e \u0939\u0948 \u0915\u093f \u0921\u0940\u090f\u092e\u0921\u0940 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 (\u0932\u0917\u092d\u0917 13-15%) \u0915\u0947 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0905\u0928\u0941\u092a\u093e\u0924 \u092e\u0947\u0902 \u0910\u0938\u0947 \u0935\u093f\u0932\u094b\u092a\u0928 \u0939\u0948\u0902 \u091c\u094b \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u091b\u094b\u0921\u093c\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0909\u0924\u094d\u0924\u0930\u0926\u093e\u092f\u0940 \u0939\u0948\u0902\u0964 \u092f\u0947 \u0935\u093f\u0932\u094b\u092a\u0928 \u0906\u092e \u0924\u094c\u0930 \u092a\u0930 \u0921\u0940\u090f\u092e\u0921\u0940 \u091c\u0940\u0928 \u0915\u0947 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u093f\u0938\u094d\u0938\u0947 \u0915\u094b \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0915\u0930\u0924\u0947 \u0939\u0948\u0902, \u091c\u093f\u0938\u0915\u0947 \u092a\u0930\u093f\u0923\u093e\u092e\u0938\u094d\u0935\u0930\u0942\u092a \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u0940 \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0937\u092e\u0924\u093e \u092e\u0947\u0902 \u0915\u092e\u0940 \u0906\u0924\u0940 \u0939\u0948\u0964 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0915\u094b \u091b\u094b\u0921\u093c\u0915\u0930, \u092f\u0947 \u0930\u094b\u0917\u0940 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0915\u093e \u090f\u0915 \u0938\u0902\u0936\u094b\u0927\u093f\u0924 \u0930\u0942\u092a \u0909\u0924\u094d\u092a\u0928\u094d\u0928 \u0915\u0930 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902 \u091c\u094b \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0915\u0940 \u0905\u0916\u0902\u0921\u0924\u093e \u0915\u094b \u0938\u0902\u0930\u0915\u094d\u0937\u093f\u0924 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948, \u092c\u0940\u092e\u093e\u0930\u0940 \u0915\u0940 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u094b \u0927\u0940\u092e\u093e \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948 \u0914\u0930 \u091c\u0940\u0935\u0928 \u0915\u0940 \u0917\u0941\u0923\u0935\u0924\u094d\u0924\u093e \u092e\u0947\u0902 \u0938\u0941\u0927\u093e\u0930 \u0915\u0930 \u0938\u0915\u0924\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u092f\u0939 \u0927\u094d\u092f\u093e\u0928 \u0930\u0916\u0928\u093e \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u0948 \u0915\u093f \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947\u0935\u0932 \u0915\u0941\u091b \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u092a\u094d\u0930\u092d\u093e\u0935\u0940 \u0939\u0948, \u0914\u0930 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u094b \u092f\u0939 \u0928\u093f\u0930\u094d\u0927\u093e\u0930\u093f\u0924 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0938\u0947 \u0917\u0941\u091c\u0930\u0928\u093e \u0939\u094b\u0917\u093e \u0915\u093f \u0915\u094d\u092f\u093e \u0935\u0947 \u090f\u0915\u094d\u0938\u0949\u0928 51-\u0906\u0927\u093e\u0930\u093f\u0924 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u0909\u092e\u094d\u092e\u0940\u0926\u0935\u093e\u0930 \u0939\u0948\u0902\u0964 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0947 \u0905\u0928\u0941\u0915\u094d\u0930\u092e\u0923 (NGS) \u0914\u0930 \u091c\u0940\u0928 \u0905\u0928\u0941\u0915\u094d\u0930\u092e\u0923 \u0924\u0915\u0928\u0940\u0915\u094b\u0902 \u0928\u0947 \u0907\u0928 \u0935\u093f\u0932\u094b\u092a\u0928\u094b\u0902 \u0915\u0940 \u092a\u0939\u091a\u093e\u0928 \u0915\u0930\u0928\u093e \u0914\u0930 \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0909\u0928\u0915\u0940 \u0905\u0928\u0941\u0915\u0942\u0932\u0924\u093e \u0915\u093e \u0906\u0915\u0932\u0928 \u0915\u0930\u0928\u093e \u0906\u0938\u093e\u0928 \u092c\u0928\u093e \u0926\u093f\u092f\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"clinical-trials-for-exon-51-skipping-therapies\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u092a\u0930\u0940\u0915\u094d\u0937\u0923<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u091c\u092c\u0915\u093f \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u092e\u0947\u0902 \u090f\u0915 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u092a\u094d\u0930\u0917\u0924\u093f \u0915\u093e \u092a\u094d\u0930\u0924\u093f\u0928\u093f\u0927\u093f\u0924\u094d\u0935 \u0915\u0930\u0924\u0940 \u0939\u0948, \u092b\u093f\u0930 \u092d\u0940 \u0915\u0908 \u091a\u0941\u0928\u094c\u0924\u093f\u092f\u093e\u0901 \u092c\u0928\u0940 \u0939\u0941\u0908 \u0939\u0948\u0902\u0964 \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0932\u093e\u092d \u0914\u0930 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930\u094b\u0902 \u0915\u0947 \u0938\u0902\u0926\u0930\u094d\u092d \u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0940 \u092a\u094d\u0930\u092d\u093e\u0935\u0936\u0940\u0932\u0924\u093e \u0915\u093e \u0905\u092d\u0940 \u092d\u0940 \u0905\u0927\u094d\u092f\u092f\u0928 \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948\u0964 \u0907\u0938\u0915\u0947 \u0905\u0924\u093f\u0930\u093f\u0915\u094d\u0924, \u0930\u094b\u0917\u0940 \u092a\u094d\u0930\u0924\u093f\u0915\u094d\u0930\u093f\u092f\u093e \u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928\u0936\u0940\u0932\u0924\u093e - \u091c\u093f\u0938\u092e\u0947\u0902 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u0915\u093e \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u093f\u0938 \u0939\u0926 \u0924\u0915 \u0939\u094b\u0924\u093e \u0939\u0948 \u0914\u0930 \u092f\u0939 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930\u094b\u0902 \u092e\u0947\u0902 \u0915\u0948\u0938\u0947 \u092a\u0930\u093f\u0935\u0930\u094d\u0924\u093f\u0924 \u0939\u094b\u0924\u093e \u0939\u0948 - \u090f\u0915 \u0938\u0924\u0924 \u091a\u0941\u0928\u094c\u0924\u0940 \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u092f\u0939\u093e\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0938\u0947 \u0938\u0902\u092c\u0902\u0927\u093f\u0924 \u0906\u0936\u093e\u091c\u0928\u0915 \u0905\u0927\u094d\u092f\u092f\u0928 \u0926\u093f\u090f \u0917\u090f \u0939\u0948\u0902:<\/strong><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"dyne-therapeutics-dyne-251-exon-51\">1\u091f\u0940\u092a\u094016\u091f\u0940 \u2013 1\u091f\u0940\u092a\u094049\u091f\u0940<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u090f\u0915 \u091c\u093e\u0902\u091a\u093e\u0924\u094d\u092e\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u0926\u0935\u093e \u0939\u0948 \u091c\u093f\u0938\u0915\u093e \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u091a\u0930\u0923 1\/2 \u0935\u0948\u0936\u094d\u0935\u093f\u0915 DELIVER \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u092e\u0947\u0902 DMD \u0938\u0947 \u092a\u0940\u0921\u093c\u093f\u0924 \u0932\u094b\u0917\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \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 (\u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/\">\u0921\u093e\u092f\u0928-251<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"jiao-tong-university-le-051\">\u091c\u093f\u0913 \u091f\u094b\u0902\u0917 \u0935\u093f\u0936\u094d\u0935\u0935\u093f\u0926\u094d\u092f\u093e\u0932\u092f - LE051<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0936\u0902\u0918\u093e\u0908 \u091c\u093f\u0913 \u091f\u094b\u0902\u0917 \u092f\u0942\u0928\u093f\u0935\u0930\u094d\u0938\u093f\u091f\u0940 \u0938\u094d\u0915\u0942\u0932 \u0911\u092b \u092e\u0947\u0921\u093f\u0938\u093f\u0928 \u0928\u0947 \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u090f\u0932\u0908051 \u0915\u0947 \u090f\u0915\u0932 \u0905\u0902\u0924\u0903\u0936\u093f\u0930\u093e \u091c\u0932\u0938\u0947\u0915 \u0915\u0940 \u0938\u0941\u0930\u0915\u094d\u0937\u093e, \u0938\u0939\u0928\u0936\u0940\u0932\u0924\u093e \u0914\u0930 \u092a\u094d\u0930\u092d\u093e\u0935\u0915\u093e\u0930\u093f\u0924\u093e \u0915\u093e \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0915\u0940 \u0918\u094b\u0937\u0923\u093e \u0915\u0940 \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\u0964 (\u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/shanghai-jiao-tong-university-school-of-medicine-begins-clinical-trials-of-le051-exon-51-skipping-therapy\/\">\u090f\u0932\u0908051<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"bio-marin-pharmaceutical-bmn-351\">\u092c\u093e\u092f\u094b\u092e\u0930\u0940\u0928 \u092b\u093e\u0930\u094d\u092e\u093e\u0938\u094d\u092f\u0941\u091f\u093f\u0915\u0932 \u2013 BMN 351<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">BMN 351, \u090f\u0915\u094d\u0938\u0949\u0928 51 \u090f\u092e\u0947\u0928\u0947\u092c\u0932 DMD \u0935\u093e\u0932\u0947 \u0932\u0921\u093c\u0915\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u090f\u0902\u091f\u0940\u0938\u0947\u0902\u0938 \u0911\u0932\u093f\u0917\u094b\u0928\u094d\u092f\u0942\u0915\u094d\u0932\u093f\u092f\u094b\u091f\u093e\u0907\u0921 (ASO) \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0940 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0939\u0948\u0964 ASO \u0910\u0938\u0940 \u0926\u0935\u093e\u090f\u0901 \u0939\u0948\u0902 \u091c\u094b \u0915\u094b\u0936\u093f\u0915\u093e\u0913\u0902 \u0926\u094d\u0935\u093e\u0930\u093e \u0915\u0941\u091b \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u092c\u0928\u093e\u0928\u0947 \u0915\u0947 \u0924\u0930\u0940\u0915\u0947 \u0915\u094b \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0915\u0930\u0924\u0940 \u0939\u0948\u0902\u0964 \u0907\u0928\u0915\u093e \u0909\u092a\u092f\u094b\u0917 \u0915\u0908 \u0905\u0932\u0917-\u0905\u0932\u0917 \u092a\u094d\u0930\u0915\u093e\u0930 \u0915\u0940 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0938\u094d\u0935\u093e\u0938\u094d\u0925\u094d\u092f \u0938\u094d\u0925\u093f\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0907\u0932\u093e\u091c \u0915\u0947 \u0932\u093f\u090f \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948\u0964 BMN 351 \u090f\u0915 \u091c\u093e\u0902\u091a \u0926\u0935\u093e \u0939\u0948, \u091c\u093f\u0938\u0915\u093e \u0905\u0930\u094d\u0925 \u0939\u0948 \u0915\u093f \u092f\u0939 \u0932\u094b\u0917\u094b\u0902 \u092e\u0947\u0902 \u0938\u0941\u0930\u0915\u094d\u0937\u093f\u0924 \u0914\u0930 \u092a\u094d\u0930\u092d\u093e\u0935\u0940 \u0928\u0939\u0940\u0902 \u092a\u093e\u0908 \u0917\u0908 \u0939\u0948 \u0914\u0930 \u0907\u0938\u0947 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923\u094b\u0902 \u0915\u0947 \u092c\u093e\u0939\u0930 \u0909\u092a\u092f\u094b\u0917 \u0915\u0947 \u0932\u093f\u090f \u0905\u0928\u0941\u092e\u094b\u0926\u093f\u0924 \u0928\u0939\u0940\u0902 \u0915\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948\u0964 (\u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/clinicaltrials.biomarin.com\/clinical-trial\/a-phase-1-2-study-to-assess-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-bmn-351-in-participants-with-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener\">\u092c\u0940\u090f\u092e\u090f\u0928 351<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"entrada-therapeutics\">Entrada Therapeutics \u2013 ENTR-601-51<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-51, \u0921\u094d\u092f\u0942\u091a\u0947\u0928 \u0915\u0947 \u0938\u093e\u0925 \u0930\u0939\u0928\u0947 \u0935\u093e\u0932\u0947 \u0932\u094b\u0917\u094b\u0902 \u0915\u0947 \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u091c\u093e\u0902\u091a \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \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, \u0915\u093e mRNA \u0930\u0940\u0921\u093f\u0902\u0917 \u092b\u094d\u0930\u0947\u092e \u0915\u094b \u092c\u0939\u093e\u0932 \u0915\u0930\u0928\u0947 \u0914\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0947 \u0905\u0928\u0941\u0935\u093e\u0926 \u0915\u0940 \u0905\u0928\u0941\u092e\u0924\u093f \u0926\u0947\u0928\u0947 \u0915\u0940 \u0907\u0938\u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u0947 \u0932\u093f\u090f \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948 \u091c\u094b \u0925\u094b\u0921\u093c\u093e \u091b\u094b\u091f\u093e \u0939\u0948 \u0932\u0947\u0915\u093f\u0928 \u0905\u092d\u0940 \u092d\u0940 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0939\u0948\u0964 (\u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/www.entradatx.com\/pipeline\" target=\"_blank\" rel=\"noopener\">ENTR-601-51<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"nippon-shinyaku-ns-051-ncnp-04\">Nippon Shinyaku \u2013 \u090f\u0928\u090f\u0938-051\/\u090f\u0928\u0938\u0940\u090f\u0928\u092a\u0940-04<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0921\u0940\u090f\u092e\u0921\u0940 \u090f\u0915 \u092a\u094d\u0930\u0917\u0924\u093f\u0936\u0940\u0932 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u0940 \u0915\u094d\u0937\u092f \u0930\u094b\u0917 \u0939\u0948 \u091c\u094b \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u092a\u094d\u0930\u094b\u091f\u0940\u0928 \u0915\u0940 \u0915\u092e\u0940 \u0915\u0947 \u0915\u093e\u0930\u0923 \u0939\u094b\u0924\u093e \u0939\u0948\u0964 \u0907\u0938\u0938\u0947 \u0915\u0902\u0915\u093e\u0932, \u0939\u0943\u0926\u092f \u0914\u0930 \u0936\u094d\u0935\u0938\u0928 \u0915\u0940 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u092e\u0947\u0902 \u0915\u092e\u091c\u093c\u094b\u0930\u0940 \u0906 \u091c\u093e\u0924\u0940 \u0939\u0948\u0964 \u0915\u0908 \u092a\u094d\u0930\u0915\u093e\u0930 \u0915\u0947 \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u093e \u0915\u093e\u0930\u0923 \u092c\u0928 \u0938\u0915\u0924\u0947 \u0939\u0948\u0902, \u0914\u0930 \u090f\u0928\u090f\u0938-051\/\u090f\u0928\u0938\u0940\u090f\u0928\u092a\u0940-04 \u0915\u094b \u0909\u0928 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0907\u0932\u093e\u091c \u0915\u0947 \u0932\u093f\u090f \u0935\u093f\u0915\u0938\u093f\u0924 \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948 \u091c\u093f\u0928\u092e\u0947\u0902 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0932\u093f\u090f \u0909\u092a\u092f\u0941\u0915\u094d\u0924 \u091c\u0940\u0928 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0915\u0940 \u092a\u0941\u0937\u094d\u091f\u093f \u0939\u094b \u091a\u0941\u0915\u0940 \u0939\u0948\u0964 \u0914\u0930 \u092a\u0922\u093c\u0947\u0902: <a href=\"https:\/\/dmdwarrior.com\/hi\/fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy\/\">FDA \u0928\u0947 NS-051\/NCNP-04 \u0915\u094b \u0905\u0928\u093e\u0925 \u0914\u0937\u0927\u093f \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u0926\u093f\u092f\u093e<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"conclusion\">\u0928\u093f\u0937\u094d\u0915\u0930\u094d\u0937<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0921\u0940\u090f\u092e\u0921\u0940 \u0915\u0947 \u0932\u093f\u090f \u090f\u0915 \u0906\u0936\u093e\u091c\u0928\u0915 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u0926\u0943\u0937\u094d\u091f\u093f\u0915\u094b\u0923 \u0915\u093e \u092a\u094d\u0930\u0924\u093f\u0928\u093f\u0927\u093f\u0924\u094d\u0935 \u0915\u0930\u0924\u0940 \u0939\u0948, \u091c\u094b \u0907\u0938 \u0924\u0915\u0928\u0940\u0915 \u0915\u0947 \u0905\u0928\u0941\u0915\u0942\u0932 \u0935\u093f\u0936\u093f\u0937\u094d\u091f \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0935\u093e\u0932\u0947 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0906\u0936\u093e \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0924\u0940 \u0939\u0948\u0964 \u091c\u092c\u0915\u093f \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0932\u093e\u092d \u0905\u092d\u0940 \u092d\u0940 \u091c\u093e\u0902\u091a \u0915\u0947 \u0905\u0927\u0940\u0928 \u0939\u0948\u0902, \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \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 \u0916\u093f\u0932\u093e\u092b \u0932\u0921\u093c\u093e\u0908 \u092e\u0947\u0902 \u090f\u0915 \u0930\u094b\u092e\u093e\u0902\u091a\u0915 \u0915\u0926\u092e \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0928\u093f\u0930\u0902\u0924\u0930 \u0905\u0928\u0941\u0938\u0902\u0927\u093e\u0928, \u0926\u0940\u0930\u094d\u0918\u0915\u093e\u0932\u093f\u0915 \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923, \u0924\u0925\u093e \u0906\u0928\u0941\u0935\u0902\u0936\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u0914\u0930 \u0935\u094d\u092f\u0915\u094d\u0924\u093f\u0917\u0924 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u092e\u0947\u0902 \u0906\u0917\u0947 \u0915\u0940 \u092a\u094d\u0930\u0917\u0924\u093f, \u0907\u0928 \u0909\u092a\u091a\u093e\u0930\u094b\u0902 \u0915\u094b \u0905\u0928\u0941\u0915\u0942\u0932\u093f\u0924 \u0915\u0930\u0928\u0947 \u0914\u0930 \u092d\u0935\u093f\u0937\u094d\u092f \u092e\u0947\u0902 \u0905\u0927\u093f\u0915 \u0921\u0940\u090f\u092e\u0921\u0940 \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0924\u0915 \u0909\u0928\u0915\u0947 \u0909\u092a\u092f\u094b\u0917 \u0915\u093e \u0935\u093f\u0938\u094d\u0924\u093e\u0930 \u0915\u0930\u0928\u0947 \u092e\u0947\u0902 \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0939\u094b\u0917\u0940\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0914\u0930 \u092a\u0922\u093c\u0947\u0902<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/hi\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0915\u0947 \u0932\u093f\u090f \u0928\u0948\u0926\u093e\u0928\u093f\u0915 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 (\u0938\u092d\u0940 \u0936\u094b\u0927\u094b\u0902 \u0915\u0940 \u0938\u0942\u091a\u0940)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Duchenne Exon 51 skipping therapies are innovative treatments aimed at addressing the genetic mutation responsible for Duchenne muscular dystrophy (DMD). By skipping exon 51 in the dystrophin gene, these therapies help produce a shorter, functional version of the dystrophin protein, potentially slowing disease progression and improving quality of life for patients. 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