{"id":4672,"date":"2025-09-29T16:14:48","date_gmt":"2025-09-29T13:14:48","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4672"},"modified":"2025-09-29T16:16:28","modified_gmt":"2025-09-29T13:16:28","slug":"dyne-therapeutics-receives-orphan-drug-designation-in-japan-for-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/hi\/dyne-therapeutics-receives-orphan-drug-designation-in-japan-for-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics \u0915\u094b \u091c\u093e\u092a\u093e\u0928 \u092e\u0947\u0902 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 \u092e\u0947\u0902 DYNE-251 \u0915\u0947 \u0932\u093f\u090f \u0905\u0928\u093e\u0925 \u0926\u0935\u093e \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u092e\u093f\u0932\u093e"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics \u0928\u0947 \u0918\u094b\u0937\u0923\u093e \u0915\u0940 \u0939\u0948 \u0915\u093f \u091c\u093e\u092a\u093e\u0928 \u092e\u0947\u0902 \u0938\u094d\u0935\u093e\u0938\u094d\u0925\u094d\u092f, \u0936\u094d\u0930\u092e \u0914\u0930 \u0915\u0932\u094d\u092f\u093e\u0923 \u092e\u0902\u0924\u094d\u0930\u093e\u0932\u092f (MHLW) \u0928\u0947 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u092e\u0938\u094d\u0915\u0941\u0932\u0930 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u0949\u092b\u0940 (DMD) \u0938\u0947 \u092a\u0940\u0921\u093c\u093f\u0924 \u0909\u0928 \u0935\u094d\u092f\u0915\u094d\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f DYNE-251 \u0915\u094b \u0905\u0928\u093e\u0925 \u0914\u0937\u0927\u093f \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u0926\u093f\u092f\u093e \u0939\u0948, \u091c\u093f\u0928\u0915\u0947 DMD \u091c\u0940\u0928 \u092e\u0947\u0902 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902 \u0914\u0930 \u091c\u094b \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947 \u0932\u093f\u090f \u0905\u0928\u0941\u0915\u0942\u0932 \u0939\u094b\u0924\u0947 \u0939\u0948\u0902\u0964 DYNE-251 \u0915\u093e \u092e\u0942\u0932\u094d\u092f\u093e\u0902\u0915\u0928 \u091a\u0932 \u0930\u0939\u0947 \u091a\u0930\u0923 1\/2 DELIVER \u0935\u0948\u0936\u094d\u0935\u093f\u0915 \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\u0964<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>&quot;\u0921\u093f\u0932\u0940\u0935\u0930 \u092a\u0930\u0940\u0915\u094d\u0937\u0923 \u092e\u0947\u0902, \u0939\u092e\u0928\u0947 \u0905\u092d\u0942\u0924\u092a\u0942\u0930\u094d\u0935 \u0914\u0930 \u0928\u093f\u0930\u0902\u0924\u0930 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930 \u0926\u0947\u0916\u093e \u0939\u0948, \u091c\u094b \u092e\u0939\u0924\u094d\u0935\u092a\u0942\u0930\u094d\u0923 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093f\u0928 \u0905\u092d\u093f\u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0926\u094d\u0935\u093e\u0930\u093e \u0938\u0902\u091a\u093e\u0932\u093f\u0924 \u0939\u0948, \u091c\u094b \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\u0940\u0921\u093c\u093f\u0924 \u0932\u094b\u0917\u094b\u0902 \u0915\u0940 \u0926\u0947\u0916\u092d\u093e\u0932 \u092e\u0947\u0902 \u092c\u0926\u0932\u093e\u0935 \u0932\u093e\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f DYNE-251 \u0915\u0940 \u0915\u094d\u0937\u092e\u0924\u093e \u0915\u094b \u0930\u0947\u0916\u093e\u0902\u0915\u093f\u0924 \u0915\u0930\u0924\u093e \u0939\u0948,&quot; \u0921\u0917 \u0915\u0947\u0930, \u090f\u092e\u0921\u0940, \u092a\u0940\u090f\u091a\u0921\u0940, \u0921\u093e\u092f\u0928 \u0915\u0947 \u092e\u0941\u0916\u094d\u092f \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u093e \u0905\u0927\u093f\u0915\u093e\u0930\u0940 \u0928\u0947 \u0915\u0939\u093e\u0964 <\/strong>&quot;\u091c\u093e\u092a\u093e\u0928 \u092e\u0947\u0902 \u0905\u092c \u0905\u0928\u093e\u0925 \u0914\u0937\u0927\u093f \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u0926\u093f\u090f \u091c\u093e\u0928\u0947 \u0915\u0947 \u0938\u093e\u0925, \u091c\u094b \u0905\u092e\u0947\u0930\u093f\u0915\u093e \u0914\u0930 \u092f\u0942\u0930\u094b\u092a \u092e\u0947\u0902 \u092e\u094c\u091c\u0942\u0926\u093e \u092a\u0926\u0928\u093e\u092e\u094b\u0902 \u0915\u093e \u092a\u0942\u0930\u0915 \u0939\u0948, DYNE-251 \u090f\u0915 \u0906\u0936\u093e\u091c\u0928\u0915 \u0905\u0917\u0932\u0940 \u092a\u0940\u0922\u093c\u0940 \u0915\u0940 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0925\u0947\u0930\u0947\u092a\u0940 \u0915\u0947 \u0930\u0942\u092a \u092e\u0947\u0902 \u0906\u0917\u0947 \u092c\u0922\u093c \u0930\u0939\u0940 \u0939\u0948\u0964 \u090f\u0915\u094d\u0938\u0949\u0928 51 \u0938\u094d\u0915\u093f\u092a\u093f\u0902\u0917 \u0915\u0947 \u0905\u0928\u0941\u0915\u0942\u0932 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0935\u093e\u0932\u0947 \u0935\u094d\u092f\u0915\u094d\u0924\u093f \u0921\u0940\u090f\u092e\u0921\u0940 \u0906\u092c\u093e\u0926\u0940 \u092e\u0947\u0902 \u0938\u092c\u0938\u0947 \u0905\u0927\u093f\u0915 \u092a\u094d\u0930\u091a\u0932\u093f\u0924 \u0914\u0930 \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0930\u0942\u092a \u0938\u0947 \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0939\u0948\u0902\u0964 \u0907\u0938 \u0905\u092a\u0942\u0930\u094d\u0923 \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u0915\u094b \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u0930\u0942\u092a \u0938\u0947 \u0938\u0902\u092c\u094b\u0927\u093f\u0924 \u0915\u0930\u0928\u0947 \u0938\u0947 \u0921\u094d\u092f\u0942\u0936\u0947\u0928 \u0938\u092e\u0941\u0926\u093e\u092f \u0915\u0947 \u0932\u093f\u090f \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930 \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u0940 \u090f\u0915 \u0925\u0947\u0930\u0947\u092a\u0940 \u0935\u093f\u0915\u0938\u093f\u0924 \u0915\u0930\u0928\u0947 \u0915\u0940 \u0939\u092e\u093e\u0930\u0940 \u092a\u094d\u0930\u0924\u093f\u092c\u0926\u094d\u0927\u0924\u093e \u0914\u0930 \u092d\u0940 \u092e\u091c\u092c\u0942\u0924 \u0939\u094b\u0924\u0940 \u0939\u0948, \u0914\u0930 \u0939\u092e \u091c\u093e\u092a\u093e\u0928 \u092e\u0947\u0902 DYNE-251 \u0915\u0947 \u0932\u093f\u090f \u0928\u093f\u092f\u093e\u092e\u0915 \u092e\u093e\u0930\u094d\u0917 \u0928\u093f\u0930\u094d\u0927\u093e\u0930\u093f\u0924 \u0915\u0930\u0928\u0947 \u0939\u0947\u0924\u0941 \u091c\u093e\u092a\u093e\u0928\u0940 \u0928\u093f\u092f\u093e\u092e\u0915\u094b\u0902 \u0915\u0947 \u0938\u093e\u0925 \u0938\u0939\u092f\u094b\u0917 \u0915\u0930\u0928\u0947 \u0915\u0947 \u0932\u093f\u090f \u0924\u0924\u094d\u092a\u0930 \u0939\u0948\u0902\u0964&quot;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u091c\u093e\u092a\u093e\u0928 \u092e\u0947\u0902 \u0905\u0928\u093e\u0925 \u0926\u0935\u093e \u0915\u094b DYNE-251 \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u0926\u093f\u092f\u093e \u0917\u092f\u093e<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u091c\u093e\u092a\u093e\u0928 \u092e\u0947\u0902, \u0905\u0928\u093e\u0925 \u0914\u0937\u0927\u093f \u0915\u093e \u0926\u0930\u094d\u091c\u093e \u0909\u0928 \u0914\u0937\u0927\u093f\u092f\u094b\u0902 \u0915\u094b \u0926\u093f\u092f\u093e \u091c\u093e\u0924\u093e \u0939\u0948 \u091c\u094b \u0926\u0947\u0936 \u092e\u0947\u0902 50,000 \u0938\u0947 \u0915\u092e \u0930\u094b\u0917\u093f\u092f\u094b\u0902 \u0915\u094b \u092a\u094d\u0930\u092d\u093e\u0935\u093f\u0924 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u0940 \u0926\u0941\u0930\u094d\u0932\u092d \u092c\u0940\u092e\u093e\u0930\u093f\u092f\u094b\u0902 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u092c\u0928\u093e\u0908 \u091c\u093e\u0924\u0940 \u0939\u0948\u0902 \u0924\u0925\u093e \u091c\u093f\u0928\u0915\u0940 \u091a\u093f\u0915\u093f\u0924\u094d\u0938\u0940\u092f \u0906\u0935\u0936\u094d\u092f\u0915\u0924\u093e \u092c\u0939\u0941\u0924 \u0905\u0927\u093f\u0915 \u0939\u094b\u0924\u0940 \u0939\u0948\u0964<\/strong> \u0932\u093e\u092d\u094b\u0902 \u092e\u0947\u0902 \u0935\u093f\u0915\u093e\u0938 \u0932\u093e\u0917\u0924 \u0915\u0947 \u0932\u093f\u090f \u0938\u092c\u094d\u0938\u093f\u0921\u0940 \u0914\u0930 \u0905\u0928\u0941\u092e\u094b\u0926\u093f\u0924 \u0939\u094b\u0928\u0947 \u092a\u0930 10 \u0935\u0930\u094d\u0937\u094b\u0902 \u0924\u0915 \u0915\u0947 \u0932\u093f\u090f \u0938\u0902\u092d\u093e\u0935\u093f\u0924 \u092c\u093e\u091c\u093e\u0930 \u0935\u093f\u0936\u093f\u0937\u094d\u091f\u0924\u093e \u0936\u093e\u092e\u093f\u0932 \u0939\u0948\u0964 DYNE-251 \u0915\u094b \u092c\u094d\u0930\u0947\u0915\u0925\u094d\u0930\u0942 \u0925\u0947\u0930\u0947\u092a\u0940 \u092a\u0926\u0928\u093e\u092e, \u092f\u0942\u090f\u0938 \u092b\u0942\u0921 \u090f\u0902\u0921 \u0921\u094d\u0930\u0917 \u090f\u0921\u092e\u093f\u0928\u093f\u0938\u094d\u091f\u094d\u0930\u0947\u0936\u0928 (FDA) \u0938\u0947 \u092b\u093e\u0938\u094d\u091f \u091f\u094d\u0930\u0948\u0915 \u0914\u0930 \u0926\u0941\u0930\u094d\u0932\u092d \u092c\u093e\u0932 \u0930\u094b\u0917 \u092a\u0926\u0928\u093e\u092e, \u0914\u0930 FDA \u0914\u0930 \u092f\u0942\u0930\u094b\u092a\u0940\u092f \u092e\u0947\u0921\u093f\u0938\u093f\u0928 \u090f\u091c\u0947\u0902\u0938\u0940 (EMA) \u0938\u0947 \u0905\u0928\u093e\u0925 \u0926\u0935\u093e \u092a\u0926\u0928\u093e\u092e \u092d\u0940 \u0926\u093f\u092f\u093e \u0917\u092f\u093e \u0939\u0948, \u091c\u094b \u0921\u0940\u090f\u092e\u0921\u0940 \u0935\u093e\u0932\u0947 \u0935\u094d\u092f\u0915\u094d\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \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 <strong>\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/hi\/dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">DYNE-251 \u0915\u0947 \u0932\u093f\u090f \u092c\u094d\u0930\u0947\u0915\u0925\u094d\u0930\u0942 \u0925\u0947\u0930\u0947\u092a\u0940 \u092a\u0926\u0928\u093e\u092e<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0915\u094d\u092f\u093e \u092e\u0947\u0930\u093e \u092c\u0947\u091f\u093e \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 \u0939\u0948?<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/hi\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\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<\/a><\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" 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=\"auto, (max-width: 847px) 100vw, 847px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251 \u0915\u0947 \u092c\u093e\u0930\u0947 \u092e\u0947\u0902<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u090f\u0915 \u0916\u094b\u091c\u0940 \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 \u0917\u094d\u0930\u0938\u094d\u0924 \u0909\u0928 \u0935\u094d\u092f\u0915\u094d\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0932\u093f\u090f \u0915\u093f\u092f\u093e \u091c\u093e \u0930\u0939\u093e \u0939\u0948 \u091c\u093f\u0928\u0915\u0947 DMD \u091c\u0940\u0928 \u092e\u0947\u0902 \u0910\u0938\u0947 \u0909\u0924\u094d\u092a\u0930\u093f\u0935\u0930\u094d\u0924\u0928 \u0939\u0948\u0902 \u091c\u094b \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\u0964 DYNE-251 \u092e\u0947\u0902 \u090f\u0915 \u092b\u0949\u0938\u094d\u092b\u094b\u0930\u094b\u0921\u093e\u092f\u092e\u093f\u0921\u0947\u091f \u092e\u0949\u0930\u094d\u092b\u094b\u0932\u093f\u0928\u094b \u0913\u0932\u093f\u0917\u094b\u092e\u0930 (PMO) \u0939\u094b\u0924\u093e \u0939\u0948 \u091c\u094b \u090f\u0915 \u090f\u0902\u091f\u0940\u091c\u0928-\u092c\u093e\u0907\u0902\u0921\u093f\u0902\u0917 \u092b\u093c\u094d\u0930\u0948\u0917\u092e\u0947\u0902\u091f (Fab) \u0938\u0947 \u0938\u0902\u092f\u0941\u0917\u094d\u092e\u093f\u0924 \u0939\u094b\u0924\u093e \u0939\u0948 \u091c\u094b \u091f\u094d\u0930\u093e\u0902\u0938\u092b\u093c\u0930\u093f\u0928 \u0930\u093f\u0938\u0947\u092a\u094d\u091f\u0930 1 (TfR1) \u0938\u0947 \u091c\u0941\u0921\u093c\u0924\u093e \u0939\u0948\u0964 \u0907\u0938\u0947 \u092e\u093e\u0902\u0938\u092a\u0947\u0936\u093f\u092f\u094b\u0902 \u0914\u0930 \u0915\u0947\u0902\u0926\u094d\u0930\u0940\u092f \u0924\u0902\u0924\u094d\u0930\u093f\u0915\u093e \u0924\u0902\u0924\u094d\u0930 (CNS) \u092e\u0947\u0902 \u0932\u0917\u092d\u0917 \u092a\u0942\u0930\u094d\u0923-\u0932\u0902\u092c\u093e\u0908 \u0935\u093e\u0932\u0947 \u0921\u093f\u0938\u094d\u091f\u094d\u0930\u094b\u092b\u093c\u093f\u0928 \u0915\u0947 \u0909\u0924\u094d\u092a\u093e\u0926\u0928 \u0915\u094b \u0938\u0915\u094d\u0937\u092e \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 \u0924\u093e\u0915\u093f \u0915\u093e\u0930\u094d\u092f\u093e\u0924\u094d\u092e\u0915 \u0938\u0941\u0927\u093e\u0930 \u092a\u094d\u0930\u0926\u093e\u0928 \u0915\u093f\u092f\u093e \u091c\u093e \u0938\u0915\u0947\u0964 DYNE-251 \u0915\u094b \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 (FDA) \u0938\u0947 \u092c\u094d\u0930\u0947\u0915\u0925\u094d\u0930\u0942 \u0925\u0947\u0930\u0947\u092a\u0940 \u092a\u0926\u0928\u093e\u092e, \u092b\u093c\u093e\u0938\u094d\u091f \u091f\u094d\u0930\u0948\u0915 \u0914\u0930 \u0926\u0941\u0930\u094d\u0932\u092d \u092c\u093e\u0932 \u0930\u094b\u0917 \u092a\u0926\u0928\u093e\u092e \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u0941\u090f \u0939\u0948\u0902, \u0938\u093e\u0925 \u0939\u0940 FDA \u0914\u0930 \u092f\u0942\u0930\u094b\u092a\u0940\u092f \u0914\u0937\u0927\u093f \u090f\u091c\u0947\u0902\u0938\u0940 (EMA) \u0938\u0947 \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 DMD \u0938\u0947 \u0917\u094d\u0930\u0938\u094d\u0924 \u0935\u094d\u092f\u0915\u094d\u0924\u093f\u092f\u094b\u0902 \u0915\u0947 \u0909\u092a\u091a\u093e\u0930 \u0915\u0947 \u0932\u093f\u090f \u0911\u0930\u094d\u092b\u093c\u0928 \u0921\u094d\u0930\u0917 \u092a\u0926\u0928\u093e\u092e \u092d\u0940 \u092a\u094d\u0930\u093e\u092a\u094d\u0924 \u0939\u0941\u0906 \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u0915\u0947 \u0905\u0924\u093f\u0930\u093f\u0915\u094d\u0924, \u0921\u093e\u092f\u0928 \u090f\u0915 DMD \u092b\u094d\u0930\u0948\u0902\u091a\u093e\u0907\u091c\u093c \u0915\u093e \u0928\u093f\u0930\u094d\u092e\u093e\u0923 \u0915\u0930 \u0930\u0939\u093e \u0939\u0948 \u0914\u0930 53, 45 \u0914\u0930 44 \u0938\u0939\u093f\u0924 \u0905\u0928\u094d\u092f \u090f\u0915\u094d\u0938\u0949\u0928 \u0915\u094b \u0932\u0915\u094d\u0937\u093f\u0924 \u0915\u0930\u0928\u0947 \u0935\u093e\u0932\u0947 \u092a\u094d\u0930\u0940\u0915\u094d\u0932\u093f\u0928\u093f\u0915\u0932 \u0915\u093e\u0930\u094d\u092f\u0915\u094d\u0930\u092e \u092d\u0940 \u091a\u0932\u093e \u0930\u0939\u093e \u0939\u0948\u0964<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0914\u0930 \u0905\u0927\u093f\u0915 \u091c\u093e\u0928\u0947\u0902<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/hi\/exon-51-skipping-therapies-dmd\/\">\u0921\u094d\u092f\u0942\u0936\u0947\u0928 \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>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. DYNE-251 is being evaluated in the ongoing Phase 1\/2 DELIVER global clinical trial. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4673,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,122,193,421,194,142],"class_list":["post-4672","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-japan"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/4672","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=4672"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/posts\/4672\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media\/4673"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/media?parent=4672"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/categories?post=4672"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/hi\/wp-json\/wp\/v2\/tags?post=4672"}],"curies":[{"name":"\u0921\u092c\u094d\u0932\u094d\u092f\u0942\u092a\u0940","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}