{"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\/ar\/exon-51-skipping-therapies-dmd\/","title":{"rendered":"\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"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51 \u0644\u062f\u0648\u0634\u064a\u0646 \u0647\u064a \u0639\u0644\u0627\u062c\u0627\u062a \u0645\u0628\u062a\u0643\u0631\u0629 \u062a\u0647\u062f\u0641 \u0625\u0644\u0649 \u0645\u0639\u0627\u0644\u062c\u0629 \u0627\u0644\u0637\u0641\u0631\u0629 \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0645\u0633\u0624\u0648\u0644\u0629 \u0639\u0646 \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a (DMD). \u0645\u0646 \u062e\u0644\u0627\u0644 \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51 \u0641\u064a \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u062a\u0633\u0627\u0639\u062f \u0647\u0630\u0647 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0639\u0644\u0649 \u0625\u0646\u062a\u0627\u062c \u0646\u0633\u062e\u0629 \u0623\u0642\u0635\u0631 \u0648\u0623\u0643\u062b\u0631 \u0641\u0639\u0627\u0644\u064a\u0629 \u0645\u0646 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0645\u0645\u0627 \u0642\u062f \u064a\u0628\u0637\u0626 \u062a\u0637\u0648\u0631 \u0627\u0644\u0645\u0631\u0636 \u0648\u064a\u062d\u0633\u0646 \u0646\u0648\u0639\u064a\u0629 \u062d\u064a\u0627\u0629 \u0627\u0644\u0645\u0631\u0636\u0649. \u064a\u0645\u0646\u062d \u0647\u0630\u0627 \u0627\u0644\u0646\u0647\u062c \u0627\u0644\u0645\u062a\u0637\u0648\u0631 \u0627\u0644\u0623\u0645\u0644 \u0644\u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u062d\u064a\u062b \u064a\u0631\u0643\u0632 \u0639\u0644\u0649 \u062a\u062d\u0633\u064a\u0646 \u0648\u0638\u064a\u0641\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0648\u0625\u0637\u0627\u0644\u0629 \u0627\u0644\u0642\u062f\u0631\u0629 \u0639\u0644\u0649 \u0627\u0644\u062d\u0631\u0643\u0629.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u062c\u062f\u0648\u0644 \u0627\u0644\u0645\u062d\u062a\u0648\u064a\u0627\u062a<\/h2><nav><ul><li><a href=\"#about-duchenne\">\u062d\u0648\u0644 \u062f\u0648\u0634\u064a\u0646<\/a><\/li><li><a href=\"#exon-51-skipping-therapies\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51<\/a><\/li><li><a href=\"#what-is-exon-51-skipping\">\u0645\u0627 \u0647\u0648 \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51\u061f<\/a><\/li><li><a href=\"#fda-approved-exon-51-skipping-therapies\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0645\u0646 FDA<\/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\">\u0639\u0645\u0644\u064a\u0627\u062a \u0627\u0644\u062d\u0630\u0641 \u0627\u0644\u0642\u0627\u0628\u0644\u0629 \u0644\u0644\u062a\u062e\u0637\u064a \u0641\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51<\/a><\/li><li><a href=\"#clinical-trials-for-exon-51-skipping-therapies\">\u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51<\/a><ul><li><a href=\"#dyne-therapeutics-dyne-251-exon-51\">Dyne Therapeutics \u2013 DYNE-251<\/a><\/li><li><a href=\"#jiao-tong-university-le-051\">\u062c\u0627\u0645\u0639\u0629 \u062c\u064a\u0627\u0648 \u062a\u0648\u0646\u063a \u2013 051 \u062c\u0646\u064a\u0647\u064b\u0627 \u0645\u0635\u0631\u064a\u064b\u0627<\/a><\/li><li><a href=\"#bio-marin-pharmaceutical-bmn-351\">\u0634\u0631\u0643\u0629 \u0628\u064a\u0648\u0645\u0627\u0631\u064a\u0646 \u0644\u0644\u0623\u062f\u0648\u064a\u0629 \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 NS-051\/NCNP-04<\/a><\/li><\/ul><\/li><li><a href=\"#conclusion\">\u062e\u0627\u062a\u0645\u0629<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"about-duchenne\">\u062d\u0648\u0644 \u062f\u0648\u0634\u064a\u0646<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a (DMD) \u0647\u0648 \u0627\u0636\u0637\u0631\u0627\u0628 \u0648\u0631\u0627\u062b\u064a \u062d\u0627\u062f \u064a\u0635\u064a\u0628 \u0627\u0644\u0623\u0648\u0644\u0627\u062f \u0628\u0634\u0643\u0644 \u0631\u0626\u064a\u0633\u064a\u060c \u0648\u064a\u0624\u062f\u064a \u0625\u0644\u0649 \u0636\u0639\u0641 \u0639\u0636\u0644\u064a \u062a\u062f\u0631\u064a\u062c\u064a\u060c \u0648\u0641\u0642\u062f\u0627\u0646 \u0644\u0644\u062d\u0631\u0643\u0629\u060c \u0648\u0641\u064a \u0627\u0644\u0646\u0647\u0627\u064a\u0629 \u0625\u0644\u0649 \u0645\u0636\u0627\u0639\u0641\u0627\u062a \u062a\u0646\u0641\u0633\u064a\u0629 \u0648\u0642\u0644\u0628\u064a\u0629. \u064a\u062d\u062f\u062b \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0646\u062a\u064a\u062c\u0629 \u0637\u0641\u0631\u0627\u062a \u0641\u064a \u062c\u064a\u0646 DMD\u060c \u0627\u0644\u0645\u0633\u0624\u0648\u0644 \u0639\u0646 \u062a\u0631\u0645\u064a\u0632 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0648\u0647\u0648 \u0628\u0631\u0648\u062a\u064a\u0646 \u0623\u0633\u0627\u0633\u064a \u064a\u064f\u062b\u0628\u0651\u062a \u0623\u063a\u0634\u064a\u0629 \u0627\u0644\u062e\u0644\u0627\u064a\u0627 \u0627\u0644\u0639\u0636\u0644\u064a\u0629. \u0628\u062f\u0648\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u062a\u0643\u0648\u0646 \u0623\u0644\u064a\u0627\u0641 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0623\u0643\u062b\u0631 \u0639\u0631\u0636\u0629 \u0644\u0644\u062a\u0644\u0641 \u0623\u062b\u0646\u0627\u0621 \u0627\u0644\u0627\u0646\u0642\u0628\u0627\u0636\u060c \u0645\u0645\u0627 \u064a\u0624\u062f\u064a \u0625\u0644\u0649 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0648\u0627\u0644\u0623\u0639\u0631\u0627\u0636 \u0627\u0644\u0645\u0645\u064a\u0632\u0629 \u0644\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"exon-51-skipping-therapies\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0627\u0644\u0637\u0641\u0631\u0629 \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0641\u064a \u062f\u0648\u0634\u064a\u0646 \u0647\u064a \u062d\u0630\u0641 \u0625\u0643\u0633\u0648\u0646\u0627\u062a \u0645\u062d\u062f\u062f\u0629 \u0641\u064a \u062c\u064a\u0646 \u062f\u0648\u0634\u064a\u0646\u060c \u0645\u0645\u0627 \u064a\u064f\u0639\u0637\u0644 \u0625\u0646\u062a\u0627\u062c \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0648\u0638\u064a\u0641\u064a. \u0623\u062d\u062f \u0623\u0633\u0627\u0644\u064a\u0628 \u0639\u0644\u0627\u062c \u062f\u0648\u0634\u064a\u0646 \u0647\u0648 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a\u060c \u0648\u0647\u064a \u0627\u0633\u062a\u0631\u0627\u062a\u064a\u062c\u064a\u0629 \u0639\u0644\u0627\u062c\u064a\u0629 \u062a\u0647\u062f\u0641 \u0625\u0644\u0649 \u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0623\u062c\u0632\u0627\u0621 \u0627\u0644\u0645\u0639\u064a\u0628\u0629 \u0645\u0646 \u0627\u0644\u062c\u064a\u0646 \u0644\u0644\u0633\u0645\u0627\u062d \u0628\u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0645\u064f\u0628\u062a\u0648\u0631 \u0648\u0644\u0643\u0646\u0647 \u063a\u064a\u0631 \u0648\u0638\u064a\u0641\u064a \u062c\u0632\u0626\u064a\u064b\u0627. \u064a\u064f\u0639\u062f \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0623\u062d\u062f \u0623\u0643\u062b\u0631 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a \u062f\u0631\u0627\u0633\u0629\u060c \u0645\u0645\u0627 \u064a\u0645\u0646\u062d \u0627\u0644\u0623\u0645\u0644 \u0644\u0645\u062c\u0645\u0648\u0639\u0629 \u0641\u0631\u0639\u064a\u0629 \u0643\u0628\u064a\u0631\u0629 \u0645\u0646 \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646.<a href=\"https:\/\/dmdwarrior.com\/ar\/what-is-exon-skipping-and-how-does-it-work\/\" data-type=\"post\" data-id=\"1445\">\u0645\u0627 \u0647\u0648 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u061f<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-exon-51-skipping\">\u0645\u0627 \u0647\u0648 \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51\u061f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u064a\u062a\u0643\u0648\u0646 \u062c\u064a\u0646 DMD \u0645\u0646 79 \u0625\u0643\u0633\u0648\u0646\u064b\u0627\u060c \u0648\u0639\u0627\u062f\u0629\u064b \u0645\u0627 \u062a\u062a\u0636\u0645\u0646 \u0627\u0644\u0637\u0641\u0631\u0627\u062a \u0641\u064a \u0647\u0630\u0627 \u0627\u0644\u062c\u064a\u0646 \u062d\u0630\u0641 \u0625\u0643\u0633\u0648\u0646\u0627\u062a \u0645\u062d\u062f\u062f\u0629. \u0639\u0627\u062f\u0629\u064b \u0645\u0627 \u062a\u0624\u062f\u064a \u0647\u0630\u0647 \u0627\u0644\u062d\u0630\u0641\u0627\u062a \u0625\u0644\u0649 \u0627\u0646\u0632\u064a\u0627\u062d \u0625\u0637\u0627\u0631 \u064a\u0645\u0646\u0639 \u0625\u0646\u062a\u0627\u062c \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u064a\u062a\u0636\u0645\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0623\u0648\u0644\u064a\u062c\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0645\u0636\u0627\u062f\u0629 \u0644\u0644\u0645\u0639\u0646\u0649 (ASOs) \u0644\u0625\u062e\u0641\u0627\u0621 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0627\u0644\u0645\u062a\u062d\u0648\u0631 \u0648\u062a\u0639\u0632\u064a\u0632 &quot;\u062a\u062e\u0637\u064a\u0647&quot; \u0623\u062b\u0646\u0627\u0621 \u0639\u0645\u0644\u064a\u0629 \u0631\u0628\u0637 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u0648\u0632\u064a (RNA). \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51\u060c \u064a\u0645\u0643\u0646 \u0631\u0628\u0637 \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a \u0627\u0644\u0644\u0627\u062d\u0642\u0629 \u0645\u0639\u064b\u0627\u060c \u0645\u0645\u0627 \u064a\u064f\u0639\u064a\u062f \u0625\u0637\u0627\u0631 \u0627\u0644\u0642\u0631\u0627\u0621\u0629 \u0648\u064a\u0633\u0645\u062d \u0628\u0625\u0646\u062a\u0627\u062c \u0634\u0643\u0644 \u0623\u0642\u0635\u0631 \u0648\u0644\u0643\u0646\u0647 \u0641\u0639\u0627\u0644 \u0645\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u0647\u062f\u0641 \u0647\u0630\u0647 \u0627\u0644\u0627\u0633\u062a\u0631\u0627\u062a\u064a\u062c\u064a\u0629 \u0625\u0644\u0649 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0648\u0638\u064a\u0641\u064a \u0627\u0644\u0630\u064a \u064a\u0645\u0643\u0646\u0647 \u0627\u0644\u062a\u0639\u0648\u064a\u0636 \u0625\u0644\u0649 \u062d\u062f \u0645\u0627 \u0639\u0646 \u0646\u0642\u0635 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0637\u0628\u064a\u0639\u064a\u060c \u0645\u0645\u0627 \u0642\u062f \u064a\u0642\u0644\u0644 \u0645\u0646 \u062a\u0637\u0648\u0631 \u0627\u0644\u0645\u0631\u0636 \u0648\u064a\u062d\u0633\u0646 \u0648\u0638\u064a\u0641\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0644\u062f\u0649 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0644\u062f\u064a\u0647\u0645 \u062d\u0630\u0641 \u0642\u0627\u0628\u0644 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. (\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <a href=\"https:\/\/dmdwarrior.com\/ar\/what-is-exon-deletion-in-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0645\u0627 \u0647\u0648 \u062d\u0630\u0641 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0641\u064a \u062f\u0648\u0634\u064a\u0646\u061f<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"fda-approved-exon-51-skipping-therapies\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0645\u0646 FDA<\/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\">\u0625\u0643\u0633\u0648\u0646\u062f\u064a\u0633 \u0665\u0661 (\u0625\u064a\u062a\u064a\u0628\u0644\u064a\u0631\u0633\u064a\u0646) \u0647\u0648 \u0623\u0648\u0644 \u0639\u0644\u0627\u062c \u0645\u0639\u062a\u0645\u062f \u0644\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0628\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u062c\u064a\u0646 FDA\u060c \u0648\u0647\u0648 \u0645\u062e\u0635\u0635 \u0644\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0644\u062f\u064a\u0647\u0645 \u0637\u0641\u0631\u0629 \u062c\u064a\u0646\u064a\u0629 \u0645\u0624\u0643\u062f\u0629 \u0641\u064a \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0648\u064a\u0645\u0643\u0646 \u0639\u0644\u0627\u062c\u0647 \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0665\u0661. \u0641\u064a \u0628\u0639\u0636 \u0627\u0644\u0645\u0631\u0636\u0649\u060c \u064a\u0633\u0627\u0639\u062f \u0647\u0630\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u0633\u0645 \u0639\u0644\u0649 \u0625\u0646\u062a\u0627\u062c \u0634\u0643\u0644 \u0623\u0642\u0635\u0631 \u0645\u0646 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. (\u0644\u0644\u0645\u0632\u064a\u062f \u0645\u0646 \u0627\u0644\u0645\u0639\u0644\u0648\u0645\u0627\u062a: <a href=\"https:\/\/dmdwarrior.com\/ar\/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\">\u0644\u0645 \u062a\u062a\u0645 \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0639\u0644\u0649 EXONDYS 51 (eteplirsen) \u0645\u0646 \u0642\u0628\u0644 \u0648\u0643\u0627\u0644\u0629 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629.<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/exondys\" target=\"_blank\" rel=\"noopener\">EMA<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"which-mutations-are-amenable-to-exon-45-skipping\">\u0639\u0645\u0644\u064a\u0627\u062a \u0627\u0644\u062d\u0630\u0641 \u0627\u0644\u0642\u0627\u0628\u0644\u0629 \u0644\u0644\u062a\u062e\u0637\u064a \u0641\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0635\u064f\u0645\u0645 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u062e\u0635\u064a\u0635\u064b\u0627 \u0644\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u062d\u0630\u0641 \u0641\u064a \u0625\u0643\u0633\u0648\u0646\u0627\u062a \u062c\u064a\u0646 \u062f\u0648\u0634\u064a\u0646 (DMD)\u060c \u0648\u0627\u0644\u0630\u064a \u064a\u064f\u0645\u0643\u0646\u0643 \u0631\u0624\u064a\u062a\u0647 \u0623\u062f\u0646\u0627\u0647. \u0647\u0630\u0647 \u0627\u0644\u062d\u0630\u0641\u0627\u062a \u0642\u0627\u0628\u0644\u0629 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51\u060c \u0625\u0630 \u064a\u064f\u0639\u064a\u062f \u062a\u062e\u0637\u064a \u0647\u0630\u0627 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0625\u0637\u0627\u0631 \u0642\u0631\u0627\u0621\u0629 \u0627\u0644\u062c\u064a\u0646\u060c \u0645\u0645\u0627 \u064a\u064f\u0624\u062f\u064a \u0625\u0644\u0649 \u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0645\u064f\u062e\u062a\u0635\u0631 \u0648\u0644\u0643\u0646\u0647 \u0641\u0639\u0627\u0644.<\/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=\"\u0627\u0644\u0637\u0641\u0631\u0627\u062a \u0648\u0627\u0644\u062d\u0630\u0641 \u0627\u0644\u0642\u0627\u0628\u0644 \u0644\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646\" 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\">\u0623\u0638\u0647\u0631\u062a \u0627\u0644\u0623\u0628\u062d\u0627\u062b \u0623\u0646 \u0646\u0633\u0628\u0629 \u0643\u0628\u064a\u0631\u0629 \u0645\u0646 \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 (\u062d\u0648\u0627\u0644\u064a 13-15%) \u0644\u062f\u064a\u0647\u0645 \u062d\u0630\u0641 \u0642\u0627\u0628\u0644 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. \u0639\u0627\u062f\u0629\u064b \u0645\u0627 \u064a\u0624\u062b\u0631 \u0647\u0630\u0627 \u0627\u0644\u062d\u0630\u0641 \u0639\u0644\u0649 \u062c\u0632\u0621 \u0623\u0633\u0627\u0633\u064a \u0645\u0646 \u062c\u064a\u0646 \u062f\u0648\u0634\u064a\u0646\u060c \u0645\u0645\u0627 \u064a\u0624\u062f\u064a \u0625\u0644\u0649 \u0641\u0642\u062f\u0627\u0646 \u0648\u0638\u064a\u0641\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51\u060c \u064a\u0645\u0643\u0646 \u0644\u0647\u0624\u0644\u0627\u0621 \u0627\u0644\u0645\u0631\u0636\u0649 \u0625\u0646\u062a\u0627\u062c \u0634\u0643\u0644 \u0645\u0639\u062f\u0651\u0644 \u0645\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0648\u0627\u0644\u0630\u064a \u0642\u062f \u064a\u062d\u0627\u0641\u0638 \u0639\u0644\u0649 \u0633\u0644\u0627\u0645\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0648\u064a\u0628\u0637\u0626 \u062a\u0637\u0648\u0631 \u0627\u0644\u0645\u0631\u0636\u060c \u0648\u064a\u062d\u0633\u0651\u0646 \u0646\u0648\u0639\u064a\u0629 \u0627\u0644\u062d\u064a\u0627\u0629.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0645\u0646 \u0627\u0644\u0645\u0647\u0645 \u0645\u0644\u0627\u062d\u0638\u0629 \u0623\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0641\u0639\u0627\u0644 \u0641\u0642\u0637 \u0641\u064a \u062d\u0627\u0644\u0627\u062a \u0637\u0641\u0631\u0627\u062a \u062c\u064a\u0646\u064a\u0629 \u0645\u0639\u064a\u0646\u0629\u060c \u0648\u064a\u062c\u0628 \u0639\u0644\u0649 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u062e\u0636\u0648\u0639 \u0644\u0627\u062e\u062a\u0628\u0627\u0631\u0627\u062a \u062c\u064a\u0646\u064a\u0629 \u0644\u062a\u062d\u062f\u064a\u062f \u0645\u062f\u0649 \u0645\u0644\u0627\u0621\u0645\u062a\u0647\u0645 \u0644\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. \u0648\u0642\u062f \u0633\u0647\u0651\u0644\u062a \u062a\u0642\u0646\u064a\u0627\u062a \u062a\u0633\u0644\u0633\u0644 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u062a\u0627\u0644\u064a (NGS) \u0648\u062a\u0633\u0644\u0633\u0644 \u0627\u0644\u062c\u064a\u0646\u0627\u062a \u062a\u062d\u062f\u064a\u062f \u0647\u0630\u0647 \u0627\u0644\u062d\u0630\u0641\u0627\u062a \u0648\u062a\u0642\u064a\u064a\u0645 \u0645\u062f\u0649 \u0645\u0644\u0627\u0621\u0645\u062a\u0647\u0627 \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"clinical-trials-for-exon-51-skipping-therapies\">\u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0631\u063a\u0645 \u0623\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u062a\u064f\u0645\u062b\u0644 \u062a\u0642\u062f\u0645\u064b\u0627 \u0645\u0644\u062d\u0648\u0638\u064b\u0627 \u0641\u064a \u0639\u0644\u0627\u062c \u062f\u0648\u0634\u064a\u0646\u060c \u0625\u0644\u0627 \u0623\u0646 \u0627\u0644\u0639\u062f\u064a\u062f \u0645\u0646 \u0627\u0644\u062a\u062d\u062f\u064a\u0627\u062a \u0644\u0627 \u062a\u0632\u0627\u0644 \u0642\u0627\u0626\u0645\u0629. \u0644\u0627 \u062a\u0632\u0627\u0644 \u0641\u0639\u0627\u0644\u064a\u0629 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0645\u0646 \u062d\u064a\u062b \u0641\u0648\u0627\u0626\u062f\u0647\u0627 \u0637\u0648\u064a\u0644\u0629 \u0627\u0644\u0623\u0645\u062f \u0648\u062a\u062d\u0633\u064a\u0646\u0627\u062a\u0647\u0627 \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0642\u064a\u062f \u0627\u0644\u062f\u0631\u0627\u0633\u0629. \u0625\u0636\u0627\u0641\u0629\u064b \u0625\u0644\u0649 \u0630\u0644\u0643\u060c \u064a\u064f\u0634\u0643\u0644 \u062a\u0628\u0627\u064a\u0646 \u0627\u0633\u062a\u062c\u0627\u0628\u0629 \u0627\u0644\u0645\u0631\u064a\u0636 - \u0628\u0645\u0627 \u0641\u064a \u0630\u0644\u0643 \u0645\u062f\u0649 \u0625\u0646\u062a\u0627\u062c \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0648\u0643\u064a\u0641\u064a\u0629 \u062a\u0631\u062c\u0645\u062a\u0647 \u0625\u0644\u0649 \u062a\u062d\u0633\u064a\u0646\u0627\u062a \u0648\u0638\u064a\u0641\u064a\u0629 - \u062a\u062d\u062f\u064a\u064b\u0627 \u0645\u0633\u062a\u0645\u0631\u064b\u0627.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0648\u0641\u064a\u0645\u0627 \u064a\u0644\u064a \u0627\u0644\u062f\u0631\u0627\u0633\u0627\u062a \u0627\u0644\u0648\u0627\u0639\u062f\u0629 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51:<\/strong><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"dyne-therapeutics-dyne-251-exon-51\">Dyne Therapeutics \u2013 DYNE-251<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u0647\u0648 \u0639\u0644\u0627\u062c \u062a\u062c\u0631\u064a\u0628\u064a \u064a\u062a\u0645 \u062a\u0642\u064a\u064a\u0645\u0647 \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2 \u0645\u0646 \u0627\u0644\u062a\u062c\u0631\u0628\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u0639\u0627\u0644\u0645\u064a\u0629 DELIVER \u0644\u0644\u0623\u0634\u062e\u0627\u0635 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u064a\u0634\u0648\u0646 \u0645\u0639 DMD \u0648\u0627\u0644\u0630\u064a\u0646 \u064a\u0645\u0643\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. (\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <a href=\"https:\/\/dmdwarrior.com\/ar\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/\">\u062f\u0627\u064a\u0646-251<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"jiao-tong-university-le-051\">\u062c\u0627\u0645\u0639\u0629 \u062c\u064a\u0627\u0648 \u062a\u0648\u0646\u063a \u2013 051 \u062c\u0646\u064a\u0647\u064b\u0627 \u0645\u0635\u0631\u064a\u064b\u0627<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0623\u0639\u0644\u0646\u062a \u0643\u0644\u064a\u0629 \u0627\u0644\u0637\u0628 \u0628\u062c\u0627\u0645\u0639\u0629 \u0634\u0646\u063a\u0647\u0627\u064a \u062c\u064a\u0627\u0648 \u062a\u0648\u0646\u063a \u0639\u0646 \u062a\u062c\u0631\u0628\u0629 \u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u062a\u0642\u064a\u064a\u0645 \u0633\u0644\u0627\u0645\u0629 \u0648\u062a\u062d\u0645\u0644 \u0648\u0641\u0639\u0627\u0644\u064a\u0629 \u062d\u0642\u0646\u0629 \u0648\u0631\u064a\u062f\u064a\u0629 \u0648\u0627\u062d\u062f\u0629 \u0645\u0646 LE051 \u0641\u064a \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a \u0627\u0644\u0642\u0627\u0628\u0644\u064a\u0646 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. (\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <a href=\"https:\/\/dmdwarrior.com\/ar\/shanghai-jiao-tong-university-school-of-medicine-begins-clinical-trials-of-le051-exon-51-skipping-therapy\/\">LE051<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"bio-marin-pharmaceutical-bmn-351\">\u0634\u0631\u0643\u0629 \u0628\u064a\u0648\u0645\u0627\u0631\u064a\u0646 \u0644\u0644\u0623\u062f\u0648\u064a\u0629 \u2013 BMN 351<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">BMN 351 \u0647\u0648 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u062c\u062f\u064a\u062f \u0645\u0646 \u0639\u0644\u0627\u062c\u0627\u062a \u0642\u0644\u064a\u0644 \u0627\u0644\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0627\u0644\u0645\u0636\u0627\u062f\u0629 \u0644\u0644\u0627\u062a\u062c\u0627\u0647 (ASO) \u0644\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 \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a \u0627\u0644\u0642\u0627\u0628\u0644 \u0644\u0644\u062a\u0623\u062b\u0631 \u0628\u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. \u062a\u0624\u062b\u0631 \u0642\u0644\u064a\u0644\u0627\u062a \u0627\u0644\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0627\u0644\u0645\u0636\u0627\u062f\u0629 \u0644\u0644\u0627\u062a\u062c\u0627\u0647 \u0639\u0644\u0649 \u0643\u064a\u0641\u064a\u0629 \u0625\u0646\u062a\u0627\u062c \u0627\u0644\u062e\u0644\u0627\u064a\u0627 \u0644\u0628\u0631\u0648\u062a\u064a\u0646\u0627\u062a \u0645\u0639\u064a\u0646\u0629\u060c \u0648\u062a\u064f\u0633\u062a\u062e\u062f\u0645 \u0644\u0639\u0644\u0627\u062c \u0623\u0646\u0648\u0627\u0639 \u0645\u062e\u062a\u0644\u0641\u0629 \u0645\u0646 \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0648\u0631\u0627\u062b\u064a\u0629. BMN 351 \u062f\u0648\u0627\u0621 \u0642\u064a\u062f \u0627\u0644\u0628\u062d\u062b\u060c \u0645\u0645\u0627 \u064a\u0639\u0646\u064a \u0623\u0646\u0647 \u0644\u0645 \u064a\u062b\u0628\u062a \u0623\u0645\u0627\u0646\u0647 \u0648\u0641\u0639\u0627\u0644\u064a\u062a\u0647 \u0639\u0644\u0649 \u0627\u0644\u0628\u0634\u0631\u060c \u0648\u0644\u0645 \u064a\u064f\u0639\u062a\u0645\u062f \u0644\u0644\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u062e\u0627\u0631\u062c \u0646\u0637\u0627\u0642 \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629. (\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <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\">\u0628\u064a \u0625\u0645 \u0625\u0646 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\u060c \u0648\u0647\u0648 \u0639\u0644\u0627\u062c \u062a\u062c\u0631\u064a\u0628\u064a \u0645\u062d\u062a\u0645\u0644 \u0644\u0639\u0644\u0627\u062c \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51\u060c \u0642\u064a\u062f \u0627\u0644\u062a\u0642\u064a\u064a\u0645 \u062d\u0627\u0644\u064a\u064b\u0627 \u0644\u0625\u0645\u0643\u0627\u0646\u064a\u0629 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0625\u0637\u0627\u0631 \u0642\u0631\u0627\u0621\u0629 mRNA \u0648\u0627\u0644\u0633\u0645\u0627\u062d \u0628\u062a\u0631\u062c\u0645\u0629 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0630\u064a \u0623\u0635\u0628\u062d \u0623\u0642\u0635\u0631 \u0642\u0644\u064a\u0644\u064b\u0627 \u0648\u0644\u0643\u0646\u0647 \u0644\u0627 \u064a\u0632\u0627\u0644 \u0641\u0639\u0627\u0644\u064b\u0627. (\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <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 NS-051\/NCNP-04<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u062f\u0648\u0634\u064a\u0646 \u0647\u0648 \u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0639\u0636\u0644\u064a \u062a\u062f\u0631\u064a\u062c\u064a \u0646\u0627\u062a\u062c \u0639\u0646 \u0646\u0642\u0635 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u064a\u0624\u062f\u064a \u0647\u0630\u0627 \u0627\u0644\u0645\u0631\u0636 \u0625\u0644\u0649 \u0636\u0639\u0641 \u0641\u064a \u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0647\u064a\u0643\u0644 \u0627\u0644\u0639\u0638\u0645\u064a \u0648\u0627\u0644\u0642\u0644\u0628 \u0648\u0627\u0644\u062c\u0647\u0627\u0632 \u0627\u0644\u062a\u0646\u0641\u0633\u064a. \u0647\u0646\u0627\u0643 \u0623\u0646\u0648\u0627\u0639 \u0639\u062f\u064a\u062f\u0629 \u0645\u0646 \u0627\u0644\u0637\u0641\u0631\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u062a\u064a \u0642\u062f \u062a\u0633\u0628\u0628 \u062f\u0648\u0634\u064a\u0646\u060c \u0648\u064a\u062c\u0631\u064a \u062a\u0637\u0648\u064a\u0631 NS-051\/NCNP-04 \u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u0644\u062f\u064a\u0647\u0645 \u0637\u0641\u0631\u0627\u062a \u062c\u064a\u0646\u064a\u0629 \u0645\u0624\u0643\u062f\u0629 \u0642\u0627\u0628\u0644\u0629 \u0644\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51. \u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <a href=\"https:\/\/dmdwarrior.com\/ar\/fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy\/\">FDA \u064a\u0645\u0646\u062d \u062a\u0635\u0646\u064a\u0641 \u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u064a\u062a\u064a\u0645 \u0644\u0640 NS-051\/NCNP-04<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"conclusion\">\u062e\u0627\u062a\u0645\u0629<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u064f\u0645\u062b\u0644 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0646\u0647\u062c\u064b\u0627 \u0639\u0644\u0627\u062c\u064a\u064b\u0627 \u0648\u0627\u0639\u062f\u064b\u0627 \u0644\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a\u060c \u0645\u0645\u0627 \u064a\u0645\u0646\u062d \u0627\u0644\u0623\u0645\u0644 \u0644\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u0637\u0641\u0631\u0627\u062a \u0645\u062d\u062f\u062f\u0629 \u0642\u0627\u0628\u0644\u0629 \u0644\u0647\u0630\u0647 \u0627\u0644\u062a\u0642\u0646\u064a\u0629. \u0648\u0628\u064a\u0646\u0645\u0627 \u0644\u0627 \u062a\u0632\u0627\u0644 \u0627\u0644\u0641\u0648\u0627\u0626\u062f \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0637\u0648\u064a\u0644\u0629 \u0627\u0644\u0645\u062f\u0649 \u0642\u064a\u062f \u0627\u0644\u0628\u062d\u062b\u060c \u062a\u064f\u0645\u062b\u0644 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u062e\u0637\u0648\u0629\u064b \u0645\u0647\u0645\u0629\u064b \u0646\u062d\u0648 \u0645\u0643\u0627\u0641\u062d\u0629 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0625\u0646 \u0627\u0644\u0627\u0633\u062a\u0645\u0631\u0627\u0631 \u0641\u064a \u0627\u0644\u0628\u062d\u062b \u0648\u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0637\u0648\u064a\u0644\u0629 \u0627\u0644\u0623\u0645\u062f \u0648\u0627\u0644\u0645\u0632\u064a\u062f \u0645\u0646 \u0627\u0644\u062a\u0642\u062f\u0645 \u0641\u064a \u0627\u0644\u0627\u062e\u062a\u0628\u0627\u0631\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0648\u0627\u0644\u0637\u0628 \u0627\u0644\u0634\u062e\u0635\u064a \u0633\u064a\u0643\u0648\u0646 \u0623\u0645\u0631\u064b\u0627 \u0628\u0627\u0644\u063a \u0627\u0644\u0623\u0647\u0645\u064a\u0629 \u0641\u064a \u062a\u062d\u0633\u064a\u0646 \u0647\u0630\u0647 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0648\u062a\u0648\u0633\u064a\u0639 \u0646\u0637\u0627\u0642 \u0627\u0633\u062a\u062e\u062f\u0627\u0645\u0647\u0627 \u0644\u0645\u0632\u064a\u062f \u0645\u0646 \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0641\u064a \u0627\u0644\u0645\u0633\u062a\u0642\u0628\u0644.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/ar\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 (\u0642\u0627\u0626\u0645\u0629 \u0628\u062c\u0645\u064a\u0639 \u0627\u0644\u0623\u0628\u062d\u0627\u062b)<\/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. This cutting-edge approach offers [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3444,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[122,193,194,77,178],"class_list":["post-3429","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-exon-skipping","tag-exon-skipping-therapies"],"subtitle":"\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 \u062f\u0648\u0634\u064a\u0646 51 \u0647\u064a \u0639\u0644\u0627\u062c\u0627\u062a \u0645\u0628\u062a\u0643\u0631\u0629 \u062a\u0647\u062f\u0641 \u0625\u0644\u0649 \u0645\u0639\u0627\u0644\u062c\u0629 \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a (DMD) \u0639\u0646 \u0637\u0631\u064a\u0642 \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646\u0627\u062a \u0645\u062d\u062f\u062f\u0629 \u0641\u064a \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u064a\u0633\u0627\u0639\u062f \u0647\u0630\u0627 \u0627\u0644\u0646\u0647\u062c \u0639\u0644\u0649 \u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0648\u0638\u064a\u0641\u064a\u060c \u0645\u0645\u0627 \u064a\u064f\u062d\u0633\u0651\u0646 \u0648\u0638\u064a\u0641\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0648\u064a\u064f\u0628\u0637\u0626 \u062a\u0637\u0648\u0631 \u0627\u0644\u0645\u0631\u0636 \u0644\u062f\u0649 \u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3429","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=3429"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3429\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3444"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3429"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3429"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3429"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}