{"id":5404,"date":"2025-12-27T16:26:26","date_gmt":"2025-12-27T13:26:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5404"},"modified":"2025-12-27T17:01:50","modified_gmt":"2025-12-27T14:01:50","slug":"one-time-gene-therapies-or-exon-skipping-therapies-which-may-be-better-for-duchenne-muscular-dystrophy-dmd-today","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/one-time-gene-therapies-or-exon-skipping-therapies-which-may-be-better-for-duchenne-muscular-dystrophy-dmd-today\/","title":{"rendered":"\u2018\u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 &quot;\u0644\u0644\u0645\u0631\u0629 \u0627\u0644\u0648\u0627\u062d\u062f\u0629&quot; \u0623\u0645 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646: \u0623\u064a\u0647\u0645\u0627 \u0642\u062f \u064a\u0643\u0648\u0646 \u0623\u0641\u0636\u0644 \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u064a\u0648\u0645\u061f"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u064a\u064f\u0639\u062f\u0651 \u062d\u0630\u0641 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0646\u0647\u062c\u064b\u0627 \u0639\u0644\u0627\u062c\u064a\u064b\u0627 \u0631\u0627\u0626\u062f\u064b\u0627 \u0644\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a (DMD)\u060c \u0648\u0647\u0648 \u0627\u0636\u0637\u0631\u0627\u0628 \u0636\u0645\u0648\u0631 \u0639\u0636\u0644\u064a \u0645\u062a\u0641\u0627\u0642\u0645 \u0646\u0627\u062a\u062c \u0639\u0646 \u0637\u0641\u0631\u0627\u062a \u0645\u0631\u0636\u064a\u0629 \u0641\u064a \u062c\u064a\u0646 DMD \u062a\u064f\u0624\u062f\u064a \u0639\u0627\u062f\u0629\u064b \u0625\u0644\u0649 \u062a\u0639\u0637\u064a\u0644 \u0625\u0637\u0627\u0631 \u0642\u0631\u0627\u0621\u0629 \u0627\u0644\u062a\u0631\u062c\u0645\u0629. \u064a\u0647\u062f\u0641 \u0647\u0630\u0627 \u0627\u0644\u0646\u0647\u062c \u0625\u0644\u0649 \u062a\u0639\u062f\u064a\u0644 \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 \u0627\u0644\u0631\u0633\u0648\u0644 \u0627\u0644\u0623\u0648\u0644\u064a (pre-mRNA) \u0644\u062c\u064a\u0646 DMD \u0644\u0625\u0639\u0627\u062f\u0629 \u0635\u064a\u0627\u063a\u0629 \u0627\u0644\u0646\u0633\u062e\u0629 \u0648\u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u0634\u0628\u0647 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0645\u062d\u0630\u0648\u0641 \u062f\u0627\u062e\u0644\u064a\u064b\u0627 \u0648\u0644\u0643\u0646\u0647 \u0648\u0638\u064a\u0641\u064a \u062c\u0632\u0626\u064a\u064b\u0627. <strong>\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/ar\/what-is-exon-skipping-and-how-does-it-work\/\" target=\"_blank\" data-type=\"post\" data-id=\"1445\" rel=\"noreferrer noopener\">\u0645\u0627 \u0647\u0648 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u061f<\/a><\/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=\"#fda-approved-exon-skipping-therapies-clinical-efficacy-is-quite-limited\">\u0625\u0646 \u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0645\u0646 \u0642\u0628\u0644 FDA \u0645\u062d\u062f\u0648\u062f\u0629 \u0644\u0644\u063a\u0627\u064a\u0629.<\/a><\/li><li><a href=\"#in-individuals-affected-by-dmd-initiating-exon-skipping-therapies-early-increases-their-effectiveness\">\u0641\u064a \u0627\u0644\u0623\u0641\u0631\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\u060c \u064a\u0624\u062f\u064a \u0627\u0644\u0628\u062f\u0621 \u0627\u0644\u0645\u0628\u0643\u0631 \u0628\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0625\u0644\u0649 \u0632\u064a\u0627\u062f\u0629 \u0641\u0639\u0627\u0644\u064a\u062a\u0647\u0627<\/a><\/li><li><a href=\"#fda-approved-dmd-therapies-have-limited-effectiveness-and-are-very-expensive\">\u062a\u062a\u0645\u064a\u0632 \u0639\u0644\u0627\u062c\u0627\u062a \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0645\u0646 \u0642\u0628\u0644 FDA \u0628\u0641\u0639\u0627\u0644\u064a\u0629 \u0645\u062d\u062f\u0648\u062f\u0629 \u0648\u0628\u062a\u0643\u0644\u0641\u0629 \u0628\u0627\u0647\u0638\u0629 \u0644\u0644\u063a\u0627\u064a\u0629<\/a><\/li><li><a href=\"#what-is-the-difference-between-pm-os-and-ppm-os\">\u0645\u0627 \u0627\u0644\u0641\u0631\u0642 \u0628\u064a\u0646 \u0645\u0643\u0627\u062a\u0628 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u0645\u0634\u0627\u0631\u064a\u0639 (PMOs) \u0648\u0645\u0643\u0627\u062a\u0628 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u0628\u0631\u0627\u0645\u062c \u0648\u0627\u0644\u0645\u0634\u0627\u0631\u064a\u0639 (PPMOs)\u061f<\/a><\/li><li><a href=\"#what-is-an-mdx-mouse-and-what-are-its-features\">\u0645\u0627 \u0647\u0648 \u0641\u0623\u0631\u0629 mdx\u060c \u0648\u0645\u0627 \u0647\u064a \u062e\u0635\u0627\u0626\u0635\u0647\u0627\u061f<\/a><\/li><li><a href=\"#efficacy-of-dystrophin-restoration-therapies\">\u0641\u0639\u0627\u0644\u064a\u0629 \u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646<\/a><ul><li><a href=\"#the-total-amount-of-dystrophin-restored\">\u0625\u062c\u0645\u0627\u0644\u064a \u0643\u0645\u064a\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0645\u0633\u062a\u0639\u0627\u062f\u0629.<\/a><\/li><li><a href=\"#the-quality-of-dystrophin-restored\">\u062a\u0645 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u062c\u0648\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646.<\/a><\/li><li><a href=\"#correct-localization-of-dystrophin-at-the-sarcolemma\">\u062a\u062d\u062f\u064a\u062f \u0645\u0648\u0642\u0639 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0634\u0643\u0644 \u0635\u062d\u064a\u062d \u0641\u064a \u063a\u0634\u0627\u0621 \u0627\u0644\u0644\u064a\u0641 \u0627\u0644\u0639\u0636\u0644\u064a.<\/a><\/li><li><a href=\"#optimal-timing-for-initiation-of-treatment\">\u0627\u0644\u062a\u0648\u0642\u064a\u062a \u0627\u0644\u0623\u0645\u062b\u0644 \u0644\u0628\u062f\u0621 \u0627\u0644\u0639\u0644\u0627\u062c.<\/a><\/li><\/ul><\/li><li><a href=\"#exon-skipping-therapies-may-also-be-implemented-as-part-of-a-combination-therapy\">\u064a\u0645\u0643\u0646 \u0623\u064a\u0636\u064b\u0627 \u062a\u0637\u0628\u064a\u0642 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0643\u062c\u0632\u0621 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0645\u0631\u0643\u0628<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"fda-approved-exon-skipping-therapies-clinical-efficacy-is-quite-limited\">\u0625\u0646 \u0627\u0644\u0641\u0639\u0627\u0644\u064a\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0645\u0646 \u0642\u0628\u0644 FDA \u0645\u062d\u062f\u0648\u062f\u0629 \u0644\u0644\u063a\u0627\u064a\u0629.<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0623\u0634\u0627\u0631\u062a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0625\u0644\u0649 \u0623\u0631\u0628\u0639\u0629 \u0639\u0644\u0627\u062c\u0627\u062a \u0645\u0639\u062a\u0645\u062f\u0629 \u0644\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 FDA \u0648\u0630\u0643\u0631\u062a \u0628\u0648\u0636\u0648\u062d \u0623\u0646 \u0641\u0639\u0627\u0644\u064a\u062a\u0647\u0627 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0645\u062d\u062f\u0648\u062f\u0629 \u0644\u0644\u063a\u0627\u064a\u0629.<\/strong> \u0648\u0644\u0644\u062a\u062d\u0642\u0642 \u0645\u0646 \u0643\u064a\u0641\u064a\u0629 \u062a\u0623\u062b\u064a\u0631 \u062a\u0648\u0642\u064a\u062a \u0627\u0644\u0639\u0644\u0627\u062c \u0639\u0644\u0649 \u0646\u062a\u0627\u0626\u062c \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u060c \u062a\u0636\u0645\u0646\u062a \u0647\u0630\u0647 \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u0639\u0644\u0645\u064a\u0629 \u062d\u0642\u0646 \u0641\u0626\u0631\u0627\u0646 mdx \u0627\u0644\u062a\u064a \u062a\u0639\u0627\u0646\u064a \u0645\u0646 \u0646\u0642\u0635 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0645\u0642\u062a\u0631\u0646\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0645\u0646 \u0628\u0628\u062a\u064a\u062f-\u0641\u0648\u0633\u0641\u0648\u0631\u0648\u062f\u064a\u0627\u0645\u064a\u062f\u0627\u062a \u0645\u0648\u0631\u0641\u0648\u0644\u064a\u0646\u0648 \u0623\u0648\u0644\u064a\u063a\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f (PPMO)\u060c \u0628\u062f\u0621\u064b\u0627 \u0645\u0646 \u0645\u0631\u0627\u062d\u0644 \u0627\u0644\u0628\u0644\u0648\u063a (12 \u0623\u0633\u0628\u0648\u0639\u064b\u0627) \u0623\u0648 \u0627\u0644\u0634\u064a\u062e\u0648\u062e\u0629 (75 \u0623\u0633\u0628\u0648\u0639\u064b\u0627)\u060c \u0648\u062c\u0645\u0639 \u0623\u0646\u0633\u062c\u0629 \u0627\u0644\u0639\u0636\u0644\u0629 \u0627\u0644\u0638\u0646\u0628\u0648\u0628\u064a\u0629 \u0627\u0644\u0623\u0645\u0627\u0645\u064a\u0629 \u0644\u0625\u062c\u0631\u0627\u0621 \u0627\u0644\u062a\u062d\u0644\u064a\u0644\u0627\u062a \u0627\u0644\u0628\u064a\u0648\u0643\u064a\u0645\u064a\u0627\u0626\u064a\u0629 \u0648\u0627\u0644\u0646\u0633\u062e\u064a\u0629.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"in-individuals-affected-by-dmd-initiating-exon-skipping-therapies-early-increases-their-effectiveness\">\u0641\u064a \u0627\u0644\u0623\u0641\u0631\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\u060c \u064a\u0624\u062f\u064a \u0627\u0644\u0628\u062f\u0621 \u0627\u0644\u0645\u0628\u0643\u0631 \u0628\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0625\u0644\u0649 \u0632\u064a\u0627\u062f\u0629 \u0641\u0639\u0627\u0644\u064a\u062a\u0647\u0627<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0628\u0644\u063a \u0645\u062a\u0648\u0633\u0637 \u062d\u0630\u0641 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 23 \u0645\u0646 \u062c\u064a\u0646 Dmd 79% \u0644\u062f\u0649 \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0628\u0627\u0644\u063a\u0629 \u064844% \u0644\u062f\u0649 \u0641\u0626\u0631\u0627\u0646 mdx \u0627\u0644\u0645\u0633\u0646\u0629 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629 \u0628\u0640 PPMO\u060c \u0628\u064a\u0646\u0645\u0627 \u0628\u0644\u063a \u0645\u062a\u0648\u0633\u0637 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 35% \u06488% \u0639\u0644\u0649 \u0627\u0644\u062a\u0648\u0627\u0644\u064a. \u0648\u0642\u062f \u0644\u0648\u062d\u0638 \u062a\u062d\u0633\u0646 \u0641\u064a \u0627\u0644\u0641\u062d\u0635 \u0627\u0644\u0646\u0633\u064a\u062c\u064a \u0627\u0644\u0645\u0631\u0636\u064a \u0641\u0642\u0637 \u0644\u062f\u0649 \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0628\u0627\u0644\u063a\u0629 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629.<\/strong> \u0623\u062f\u0649 \u0627\u0644\u0639\u0644\u0627\u062c \u0628\u0640 PPMO \u0641\u064a \u0641\u0626\u0631\u0627\u0646 mdx \u0627\u0644\u0628\u0627\u0644\u063a\u0629 \u0625\u0644\u0649 \u062a\u062d\u0648\u0644 \u0648\u0627\u0633\u0639 \u0627\u0644\u0646\u0637\u0627\u0642 \u0641\u064a \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0627\u0644\u062c\u064a\u0646\u064a \u0646\u062d\u0648 \u0627\u0644\u0646\u0645\u0637 \u0627\u0644\u0628\u0631\u064a\u060c \u0628\u064a\u0646\u0645\u0627 \u0623\u0633\u0641\u0631 \u0627\u0644\u0639\u0644\u0627\u062c \u0641\u064a \u0627\u0644\u0641\u0626\u0631\u0627\u0646 \u0627\u0644\u0645\u0633\u0646\u0629 \u0639\u0646 \u062a\u063a\u064a\u064a\u0631\u0627\u062a \u0637\u0641\u064a\u0641\u0629 \u0641\u064a \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0627\u0644\u062c\u064a\u0646\u064a\u060c \u0645\u0645\u0627 \u064a\u0634\u064a\u0631 \u0625\u0644\u0649 \u0623\u0646 \u0627\u0644\u062a\u062f\u062e\u0644 \u0627\u0644\u0645\u062a\u0623\u062e\u0631 \u063a\u064a\u0631 \u0641\u0639\u0627\u0644 \u0641\u064a \u0639\u0643\u0633 \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0645\u0635\u0627\u062d\u0628\u0629 \u0644\u0644\u0645\u0631\u0636 \u0639\u0644\u0649 \u0627\u0644\u0631\u063a\u0645 \u0645\u0646 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0645\u0633\u062a\u0648\u0649 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0634\u0643\u0644 \u0637\u0641\u064a\u0641. \u0648\u0642\u062f \u062a\u0645 \u062a\u0635\u062d\u064a\u062d \u0627\u062e\u062a\u0644\u0627\u0644 \u062a\u0648\u0627\u0632\u0646 \u0646\u0633\u062e \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0641\u0642\u0637 \u0641\u064a \u0641\u0626\u0631\u0627\u0646 mdx \u0627\u0644\u0628\u0627\u0644\u063a\u0629 \u0627\u0644\u0645\u0639\u0627\u0644\u062c\u0629. \u0648\u064a\u064f\u0642\u062f\u0645 \u0627\u0632\u062f\u064a\u0627\u062f \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0639\u0646 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u0648\u0632\u064a \u0627\u0644\u0645\u064a\u0643\u0631\u0648\u064a miR-31-5p\u060c \u0627\u0644\u0630\u064a \u062a\u0636\u0627\u0639\u0641 \u0628\u0634\u0643\u0644 \u0623\u0643\u0628\u0631 \u0641\u064a \u0639\u0636\u0644\u0627\u062a \u0641\u0626\u0631\u0627\u0646 mdx \u0627\u0644\u0645\u0633\u0646\u0629\u060c \u062a\u0641\u0633\u064a\u0631\u064b\u0627 \u0622\u0644\u064a\u064b\u0627 \u0645\u062d\u062a\u0645\u0644\u0627\u064b. <strong>\u0641\u064a \u0627\u0644\u062e\u062a\u0627\u0645\u060c \u064a\u0643\u0648\u0646 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0628\u0648\u0633\u0627\u0637\u0629 PPMO \u0623\u0643\u062b\u0631 \u0641\u0639\u0627\u0644\u064a\u0629 \u0628\u0634\u0643\u0644 \u0643\u0628\u064a\u0631 \u0639\u0646\u062f \u0628\u062f\u0621 \u0627\u0644\u0639\u0645\u0644\u064a\u0629 \u0641\u064a \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0645\u0635\u0627\u0628\u0629 \u0628\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0644\u062f\u0649 \u0627\u0644\u0628\u0627\u0644\u063a\u064a\u0646 \u0645\u0642\u0627\u0631\u0646\u0629 \u0628\u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0645\u0635\u0627\u0628\u0629 \u0628\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0644\u062f\u0649 \u0643\u0628\u0627\u0631 \u0627\u0644\u0633\u0646\u060c \u0645\u0645\u0627 \u064a\u062f\u0639\u0645 \u0627\u0644\u062a\u062f\u062e\u0644 \u0627\u0644\u0639\u0644\u0627\u062c\u064a \u0627\u0644\u0645\u0628\u0643\u0631 \u0644\u062f\u0649 \u0627\u0644\u0623\u0641\u0631\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.<\/strong> &gt;&gt; <strong>\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/Duchenne-Population-Potentially-Amenable-to-Exon-Skipping-01.04.25.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">\u0645\u062c\u0645\u0648\u0639\u0629 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0645\u062d\u062a\u0645\u0644\u0629 \u0627\u0644\u0642\u0627\u0628\u0644\u0629 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"fda-approved-dmd-therapies-have-limited-effectiveness-and-are-very-expensive\">\u062a\u062a\u0645\u064a\u0632 \u0639\u0644\u0627\u062c\u0627\u062a \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0645\u0646 \u0642\u0628\u0644 FDA \u0628\u0641\u0639\u0627\u0644\u064a\u0629 \u0645\u062d\u062f\u0648\u062f\u0629 \u0648\u0628\u062a\u0643\u0644\u0641\u0629 \u0628\u0627\u0647\u0638\u0629 \u0644\u0644\u063a\u0627\u064a\u0629<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0634\u0647\u062f\u062a \u0627\u0644\u0633\u0646\u0648\u0627\u062a \u0627\u0644\u0623\u062e\u064a\u0631\u0629 \u062a\u0642\u062f\u0645\u0627\u064b \u0645\u0644\u062d\u0648\u0638\u0627\u064b \u0641\u064a \u0645\u062c\u0627\u0644 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062a\u062c\u0631\u064a\u0628\u064a\u0629 \u0644\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a. \u0648\u0642\u062f \u0627\u0633\u062a\u064f\u062e\u062f\u0645\u062a \u0627\u0633\u062a\u0631\u0627\u062a\u064a\u062c\u064a\u0627\u062a \u0645\u062a\u0646\u0648\u0639\u0629 \u0644\u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u0639\u0644\u0649 \u0633\u0628\u064a\u0644 \u0627\u0644\u0645\u062b\u0627\u0644\u060c \u064a\u0645\u0643\u0646 \u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u062a\u0642\u0646\u064a\u0629 \u062d\u0630\u0641 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0628\u0648\u0633\u0627\u0637\u0629 \u0642\u0644\u064a\u0644 \u0627\u0644\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0627\u0644\u0645\u0636\u0627\u062f (ASO) \u0644\u062a\u0639\u062f\u064a\u0644 \u0639\u0645\u0644\u064a\u0629 \u0627\u0644\u062a\u0636\u0641\u064a\u0631 \u0627\u0644\u0628\u062f\u064a\u0644 \u0628\u062d\u064a\u062b \u064a\u062a\u0645 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0625\u0637\u0627\u0631 \u0642\u0631\u0627\u0621\u0629 \u062a\u0631\u062c\u0645\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. <strong>\u062d\u0635\u0644\u062a \u0623\u0631\u0628\u0639\u0629 \u0623\u062f\u0648\u064a\u0629 \u0645\u0646 \u0646\u0648\u0639 \u0623\u0648\u0644\u064a\u063a\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0645\u0636\u0627\u062f\u0629 \u0644\u0644\u062c\u064a\u0646\u0627\u062a \u0639\u0644\u0649 \u0645\u0648\u0627\u0641\u0642\u0629 \u0633\u0631\u064a\u0639\u0629 \u0645\u0646 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u063a\u0630\u0627\u0621 \u0648\u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u0623\u0645\u0631\u064a\u0643\u064a\u0629 (FDA) \u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0623\u0637\u0641\u0627\u0644 \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 (DMD) (Eteplirsen\u060c \u0641\u064a\u0644\u062a\u0648\u0644\u0627\u0631\u0633\u0646\u060c \u063a\u0648\u0644\u0648\u062f\u064a\u0631\u064a\u0633\u0646\u060c \u0648\u0643\u0627\u0633\u064a\u0645\u064a\u0631\u0633\u0646). \u0648\u0631\u063a\u0645 \u0623\u0646 \u0647\u0630\u0627 \u064a\u064f\u0639\u062f \u0625\u0646\u062c\u0627\u0632\u064b\u0627 \u0647\u0627\u0645\u064b\u0627 \u0644\u0623\u0628\u062d\u0627\u062b \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a \u0648\u0645\u062c\u062a\u0645\u0639\u0627\u062a \u0627\u0644\u0645\u0631\u0636\u0649\u060c \u0625\u0644\u0627 \u0623\u0646 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u062a\u064a \u062a\u062d\u0642\u0642\u0647\u0627 \u0647\u0630\u0647 \u0627\u0644\u0645\u0631\u0643\u0628\u0627\u062a \u0636\u0626\u064a\u0644\u0629\u060c \u0648\u0641\u0648\u0627\u0626\u062f\u0647\u0627 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0645\u062d\u0644 \u062c\u062f\u0644.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0648\u0628\u0627\u0644\u0645\u062b\u0644\u060c \u062a\u0645\u062a \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0639\u0644\u0649 \u0623\u0648\u0644 \u0639\u0644\u0627\u062c \u062c\u064a\u0646\u064a \u0644\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a (Elevidys) \u0641\u064a \u0639\u0627\u0645 2023\u060c \u0639\u0644\u0649 \u0627\u0644\u0631\u063a\u0645 \u0645\u0646 \u0641\u0634\u0644\u0647 \u0641\u064a \u062a\u062d\u0642\u064a\u0642 \u0647\u062f\u0641\u0647 \u0627\u0644\u0633\u0631\u064a\u0631\u064a \u0641\u064a \u0627\u0644\u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u062b\u0627\u0644\u062b\u0629 \u0645\u0646 \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629. \u0639\u0644\u0627\u0648\u0629 \u0639\u0644\u0649 \u0630\u0644\u0643\u060c \u0623\u064f\u062b\u064a\u0631\u062a \u062a\u0633\u0627\u0624\u0644\u0627\u062a \u062d\u0648\u0644 \u0633\u0644\u0627\u0645\u0629 Elevidys (\u0648\u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u062c\u0631\u0639\u0627\u062a \u0639\u0627\u0644\u064a\u0629 \u0628\u0634\u0643\u0644 \u0639\u0627\u0645) \u0628\u0639\u062f \u0648\u0641\u0627\u0629 \u0627\u0644\u0639\u062f\u064a\u062f \u0645\u0646 \u0627\u0644\u0645\u0631\u0636\u0649. <strong>\u0627\u0643\u062a\u0634\u0641 \u0627\u0644\u0645\u0632\u064a\u062f:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/ar\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0644\u0645\u0627\u0630\u0627 \u0644\u0645 \u062a\u062a\u0645 \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0639\u0644\u0649 Elevidys \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 (EMA)\u061f<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-the-difference-between-pm-os-and-ppm-os\">\u0645\u0627 \u0627\u0644\u0641\u0631\u0642 \u0628\u064a\u0646 \u0645\u0643\u0627\u062a\u0628 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u0645\u0634\u0627\u0631\u064a\u0639 (PMOs) \u0648\u0645\u0643\u0627\u062a\u0628 \u0625\u062f\u0627\u0631\u0629 \u0627\u0644\u0628\u0631\u0627\u0645\u062c \u0648\u0627\u0644\u0645\u0634\u0627\u0631\u064a\u0639 (PPMOs)\u061f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u064f\u0639\u062f\u0651 \u062c\u0645\u064a\u0639 \u062c\u0632\u064a\u0626\u0627\u062a \u0627\u0644\u0623\u0648\u0644\u064a\u063a\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0627\u0644\u0645\u0648\u0631\u0641\u0648\u0644\u064a\u0646\u064a\u0629 \u0627\u0644\u0641\u0648\u0633\u0641\u0648\u0631\u0648\u062f\u064a\u0627\u0645\u064a\u062f\u0627\u062a\u064a\u0629 (PMOs) \u0645\u0646 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u062d\u0627\u0644\u064a \u0645\u0646 \u0645\u0636\u0627\u062f\u0627\u062a \u0627\u0644\u0623\u0648\u0644\u064a\u063a\u0648\u0646\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f\u0627\u062a \u0627\u0644\u0645\u064f\u0639\u062f\u0651\u0644\u0629 \u0644\u062d\u0630\u0641 \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a. \u0644\u0627 \u062a\u0648\u062c\u062f \u0647\u0630\u0647 \u0627\u0644\u062a\u0631\u0643\u064a\u0628\u0629 \u0627\u0644\u0643\u064a\u0645\u064a\u0627\u0626\u064a\u0629 \u0641\u064a \u0627\u0644\u0637\u0628\u064a\u0639\u0629\u060c \u0648\u062a\u064f\u0639\u062a\u0628\u0631 \u0622\u0645\u0646\u0629 \u0639\u0644\u0649 \u0646\u0637\u0627\u0642 \u0648\u0627\u0633\u0639. \u0645\u0639 \u0630\u0644\u0643\u060c \u0641\u0625\u0646\u0651 \u062a\u0648\u0635\u064a\u0644 PMOs \u0625\u0644\u0649 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0647\u064a\u0643\u0644\u064a\u0629 (\u0648\u062e\u0627\u0635\u0629\u064b \u0639\u0636\u0644\u0629 \u0627\u0644\u0642\u0644\u0628) \u0645\u062d\u062f\u0648\u062f \u0644\u0644\u063a\u0627\u064a\u0629\u060c \u0645\u0645\u0627 \u062d\u0641\u0651\u0632 \u0627\u0644\u0628\u062d\u062b \u0639\u0646 \u062c\u0632\u064a\u0626\u0627\u062a \u0645\u064f\u0633\u0627\u0639\u062f\u0629 \u0639\u0644\u0649 \u0627\u0644\u062a\u0648\u0635\u064a\u0644 \u062a\u064f\u0639\u0632\u0651\u0632 \u0641\u0639\u0627\u0644\u064a\u062a\u0647\u0627. \u0648\u0642\u062f \u0627\u0633\u062a\u0643\u0634\u0641 \u0627\u0644\u0639\u0644\u0645\u0627\u0621 \u0639\u0644\u0649 \u0646\u0637\u0627\u0642 \u0648\u0627\u0633\u0639 \u0627\u0633\u062a\u062e\u062f\u0627\u0645 PMOs \u0627\u0644\u0645\u064f\u0642\u062a\u0631\u0646\u0629 \u0628\u0627\u0644\u0628\u0628\u062a\u064a\u062f\u0627\u062a (PPMOs) \u0644\u0647\u0630\u0647 \u0627\u0644\u0623\u063a\u0631\u0627\u0636. <strong>\u062a\u0639\u062a\u0628\u0631 PPMOs \u0623\u0643\u062b\u0631 \u0641\u0639\u0627\u0644\u064a\u0629 \u0628\u0643\u062b\u064a\u0631 \u0645\u0646 PMOs \u063a\u064a\u0631 \u0627\u0644\u0645\u0642\u062a\u0631\u0646\u0629\u060c \u0648\u062a\u0638\u0647\u0631 \u0646\u0634\u0627\u0637 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0641\u064a \u0623\u0646\u0633\u062c\u0629 \u0627\u0644\u0642\u0644\u0628.<\/strong> \u064a\u062c\u0631\u064a \u062d\u0627\u0644\u064a\u064b\u0627 \u062a\u0637\u0648\u064a\u0631 PPMOs \u062a\u062c\u0627\u0631\u064a\u064b\u0627 \u0645\u0646 \u0642\u0628\u0644 \u0634\u0631\u0643\u062a\u064a PepGen Ltd \u0648Entrada Therapeutics. \u0648\u0628\u0627\u0644\u0645\u062b\u0644\u060c \u062a\u0639\u0645\u0644 \u0634\u0631\u0643\u062a\u0627 Avidity Biosciences \u0648Dyne Therapeutics \u0639\u0644\u0649 \u062a\u0637\u0648\u064a\u0631 \u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u0623\u062c\u0633\u0627\u0645 \u0627\u0644\u0645\u0636\u0627\u062f\u0629 \u0644\u0640 PMO \u0648\u062c\u0632\u064a\u0626\u0627\u062a PMO-FAb \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a. <strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/ar\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0645\u0646 \u0627\u0644\u062c\u064a\u0644 \u0627\u0644\u0642\u0627\u062f\u0645<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-an-mdx-mouse-and-what-are-its-features\">\u0645\u0627 \u0647\u0648 \u0641\u0623\u0631\u0629 mdx\u060c \u0648\u0645\u0627 \u0647\u064a \u062e\u0635\u0627\u0626\u0635\u0647\u0627\u061f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u064f\u0639\u062f \u0641\u0623\u0631 mdx \u0627\u0644\u0646\u0645\u0648\u0630\u062c \u0627\u0644\u0641\u0623\u0631\u064a \u0627\u0644\u0623\u0643\u062b\u0631 \u0627\u0633\u062a\u062e\u062f\u0627\u0645\u064b\u0627 \u0644\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a\u060c \u0648\u0627\u0644\u0630\u064a \u064a\u062d\u0645\u0644 \u0643\u0648\u062f\u0648\u0646 \u0625\u0646\u0647\u0627\u0621 \u0645\u0628\u0643\u0631 \u0641\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 23 \u0645\u0646 \u062c\u064a\u0646 Dmd\u060c \u0648\u0627\u0644\u0630\u064a \u064a\u0624\u062f\u064a \u062a\u062e\u0637\u064a\u0647 \u0625\u0644\u0649 \u0636\u0645\u0648\u0631 \u0639\u0636\u0644\u064a \u0645\u0631\u062a\u0628\u0637 \u0628\u0627\u0644\u0643\u0631\u0648\u0645\u0648\u0633\u0648\u0645 X \u0648\u0627\u0644\u0630\u064a \u064a\u064f\u0638\u0647\u0631 \u0628\u0639\u0636 \u062c\u0648\u0627\u0646\u0628 \u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a.<\/strong> \u0641\u064a \u0647\u0630\u0647 \u0627\u0644\u062f\u0631\u0627\u0633\u0629\u060c \u0623\u062c\u0631\u0649 \u0627\u0644\u0639\u0644\u0645\u0627\u0621 \u0633\u0627\u0628\u0642\u064b\u0627 \u0645\u062c\u0645\u0648\u0639\u0629 \u0645\u062a\u0646\u0648\u0639\u0629 \u0645\u0646 \u062a\u062d\u0644\u064a\u0644\u0627\u062a \u0627\u0644\u0623\u0648\u0645\u064a\u0643\u0633 \u0641\u064a \u0647\u0630\u0627 \u0627\u0644\u0641\u0623\u0631 \u0645\u0639 \u0627\u0644\u062a\u0631\u0643\u064a\u0632 \u0639\u0644\u0649 \u0622\u062b\u0627\u0631 \u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0628\u0645\u0627 \u0641\u064a \u0630\u0644\u0643 \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0627\u0644\u062c\u064a\u0646\u064a \/ \u0645\u0635\u0641\u0648\u0641\u0629 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u064a \u0627\u0644\u0645\u064a\u0643\u0631\u0648\u064a\u060c \u0648\u062a\u0633\u0644\u0633\u0644 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u064a \u0627\u0644\u0635\u063a\u064a\u0631\u060c \u0648\u0627\u0644\u0628\u0631\u0648\u062a\u064a\u0648\u0645\u064a\u0627\u062a \u0627\u0644\u0642\u0627\u0626\u0645\u0629 \u0639\u0644\u0649 \u0642\u064a\u0627\u0633 \u0627\u0644\u0637\u064a\u0641 \u0627\u0644\u0643\u062a\u0644\u064a \u0639\u0627\u0644\u064a \u0627\u0644\u062f\u0642\u0629.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"efficacy-of-dystrophin-restoration-therapies\">\u0641\u0639\u0627\u0644\u064a\u0629 \u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-total-amount-of-dystrophin-restored\">\u0625\u062c\u0645\u0627\u0644\u064a \u0643\u0645\u064a\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0645\u0633\u062a\u0639\u0627\u062f\u0629.<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0645\u0646 \u0627\u0644\u0645\u0633\u0644\u0651\u0645 \u0628\u0647 \u0639\u0644\u0649 \u0646\u0637\u0627\u0642 \u0648\u0627\u0633\u0639 \u0623\u0646\u0647 \u0643\u0644\u0645\u0627 \u0632\u0627\u062f\u062a \u0643\u0645\u064a\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u062a\u064a \u064a\u0645\u0643\u0646 \u0627\u0633\u062a\u0639\u0627\u062f\u062a\u0647\u0627\u060c \u0643\u0627\u0646 \u0630\u0644\u0643 \u0623\u0641\u0636\u0644. \u0648\u062a\u0634\u064a\u0631 \u0623\u062f\u0644\u0629 \u0645\u062a\u0639\u062f\u062f\u0629 \u0625\u0644\u0649 \u0623\u0646 \u0645\u0633\u062a\u0648\u0649 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0635\u062d\u064a \u0627\u0644\u0630\u064a \u064a\u0628\u0644\u063a \u062d\u0648\u0627\u0644\u064a 10% \u064a\u0645\u062b\u0644 \u0639\u062a\u0628\u0629 \u0645\u0639\u0642\u0648\u0644\u0629 \u0644\u062a\u062d\u0642\u064a\u0642 \u0641\u0627\u0626\u062f\u0629 \u0639\u0644\u0627\u062c\u064a\u0629.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-quality-of-dystrophin-restored\">\u062a\u0645 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u062c\u0648\u062f\u0629 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646.<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u064a\u0639\u062a\u0645\u062f \u0643\u0644 \u0645\u0646 \u062a\u0635\u062d\u064a\u062d \u0627\u0644\u0631\u0628\u0637 \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0648\u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0644\u0644\u0645\u064a\u0643\u0631\u0648\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0639\u0644\u0649 \u062a\u0648\u0644\u064a\u062f \u0628\u0631\u0648\u062a\u064a\u0646\u0627\u062a \u0634\u0628\u0647 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0645\u062d\u0630\u0648\u0641\u0629 \u062f\u0627\u062e\u0644\u064a\u064b\u0627 \u062a\u0634\u0628\u0647 \u062a\u0644\u0643 \u0627\u0644\u0645\u0648\u062c\u0648\u062f\u0629 \u0641\u064a \u0628\u0639\u0636 \u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u0628\u064a\u0643\u0631 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u0648\u0627\u0644\u062a\u064a \u062a\u0631\u062a\u0628\u0637 \u0628\u0623\u0645\u0631\u0627\u0636 \u062e\u0641\u064a\u0641\u0629. <strong>\u0645\u0639 \u0630\u0644\u0643\u060c \u0645\u0646 \u063a\u064a\u0631 \u0627\u0644\u0645\u0631\u062c\u062d \u0623\u0646 \u062a\u0639\u0648\u0636 \u0647\u0630\u0647 \u0627\u0644\u0628\u0631\u0648\u062a\u064a\u0646\u0627\u062a \u0627\u0644\u0634\u0628\u064a\u0647\u0629 \u0628\u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0634\u0643\u0644 \u0643\u0627\u0645\u0644 \u0639\u0646 \u0641\u0642\u062f\u0627\u0646 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644. \u0648\u0647\u0646\u0627\u0643 \u0625\u062f\u0631\u0627\u0643 \u0645\u062a\u0632\u0627\u064a\u062f \u0644\u0623\u0647\u0645\u064a\u0629 \u0648\u062c\u0648\u062f \u0628\u0631\u0648\u062a\u064a\u0646\u0627\u062a \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644\u0629 \u0627\u0644\u0637\u0648\u0644\u060c \u0648\u0647\u0648 \u0645\u0627 \u064a\u0645\u062b\u0644 \u062a\u062d\u062f\u064a\u064b\u0627 \u0646\u0638\u0631\u064b\u0627 \u0644\u0623\u0646 \u062d\u062c\u0645 \u0625\u0637\u0627\u0631 \u0627\u0644\u0642\u0631\u0627\u0621\u0629 \u0627\u0644\u0645\u0641\u062a\u0648\u062d \u0644\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 (\u062d\u0648\u0627\u0644\u064a 11 \u0643\u064a\u0644\u0648\u0628\u0627\u064a\u062a) \u064a\u062a\u062c\u0627\u0648\u0632 \u0633\u0639\u0629 \u0627\u0644\u062a\u0639\u0628\u0626\u0629 \u0644\u0646\u0627\u0642\u0644\u0627\u062a \u0627\u0644\u0641\u064a\u0631\u0648\u0633\u0627\u062a \u0627\u0644\u063a\u062f\u064a\u0629 \u0627\u0644\u0645\u0631\u062a\u0628\u0637\u0629 (\u062d\u0648\u0627\u0644\u064a 4.6 \u0643\u064a\u0644\u0648\u0628\u0627\u064a\u062a).<\/strong> \u0644\u0642\u062f \u062b\u0628\u062a\u062a \u0641\u0639\u0627\u0644\u064a\u0629 \u0627\u0633\u062a\u0639\u0627\u062f\u0629 \u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644 \u0644\u062f\u0649 \u0627\u0644\u0645\u0634\u0627\u0631\u0643\u064a\u0646 \u0641\u064a \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u0628\u0634\u0631\u064a\u0629 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u062a\u0642\u0646\u064a\u0629 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\u0628\u0627\u0633\u062a\u062e\u062f\u0627\u0645 \u0646\u0648\u0627\u0642\u0644 \u0630\u0627\u062a \u0642\u062f\u0631\u0627\u062a \u062a\u063a\u0644\u064a\u0641 \u0623\u0639\u0644\u0649.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"correct-localization-of-dystrophin-at-the-sarcolemma\">\u062a\u062d\u062f\u064a\u062f \u0645\u0648\u0642\u0639 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0634\u0643\u0644 \u0635\u062d\u064a\u062d \u0641\u064a \u063a\u0634\u0627\u0621 \u0627\u0644\u0644\u064a\u0641 \u0627\u0644\u0639\u0636\u0644\u064a.<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u0623\u0638\u0647\u0631\u062a \u0647\u0630\u0647 \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0623\u0646 \u0627\u0644\u062a\u0648\u0632\u064a\u0639 \u0627\u0644\u0645\u0646\u062a\u0638\u0645 \u0644\u0628\u0631\u0648\u062a\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0639\u0644\u0649 \u0637\u0648\u0644 \u063a\u0634\u0627\u0621 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<strong>\u062a\u062f\u0639\u0645 \u0647\u0630\u0647 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0641\u0643\u0631\u0629 \u0623\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0645\u0628\u0643\u0631 \u0642\u062f \u064a\u0643\u0648\u0646 \u0645\u0641\u064a\u062f\u064b\u0627 \u0641\u064a \u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"exon-skipping-therapies-may-also-be-implemented-as-part-of-a-combination-therapy\">\u064a\u0645\u0643\u0646 \u0623\u064a\u0636\u064b\u0627 \u062a\u0637\u0628\u064a\u0642 \u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0643\u062c\u0632\u0621 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0645\u0631\u0643\u0628<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u0645\u0643\u0646 \u0627\u0644\u0628\u062f\u0621 \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0644\u062f\u0649 \u0627\u0644\u0623\u0648\u0644\u0627\u062f \u0627\u0644\u0635\u063a\u0627\u0631 \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 \u0628\u0647\u062f\u0641 \u0627\u0644\u062d\u0641\u0627\u0638 \u0639\u0644\u0649 \u0648\u0638\u0627\u0626\u0641 \u0639\u0636\u0644\u0627\u062a\u0647\u0645 \u062d\u062a\u0649 \u0633\u0646 \u064a\u0643\u0648\u0646 \u0641\u064a\u0647\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0628\u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0622\u0645\u0646\u0627\u064b \u0648\u0645\u0646\u0627\u0633\u0628\u0627\u064b.<\/strong> \u0641\u064a \u0627\u0644\u0648\u0627\u0642\u0639\u060c \u062a\u062f\u0639\u0645 \u0627\u0644\u0623\u062f\u0644\u0629 \u0645\u0627 \u0642\u0628\u0644 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0648\u062c\u0648\u062f \u062a\u0623\u062b\u064a\u0631 \u062a\u0622\u0632\u0631\u064a \u0644\u062f\u0645\u062c \u062a\u0642\u0646\u064a\u0629 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0628\u0648\u0633\u0627\u0637\u0629 PMO \u0645\u0639 \u062a\u0642\u0646\u064a\u0629 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0628\u0648\u0633\u0627\u0637\u0629 AAV-U7 \u0623\u0648 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0628\u0627\u0633\u062a\u062e\u062f\u0627\u0645 AAV-microdystrophin. \u062a\u064f\u0636\u064a\u0641 \u0647\u0630\u0647 \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0625\u0644\u0649 \u0627\u0644\u0646\u0642\u0627\u0634 \u0627\u0644\u062f\u0627\u0626\u0631 \u062d\u0648\u0644 \u0627\u0644\u062a\u0648\u0642\u064a\u062a \u0627\u0644\u0623\u0645\u062b\u0644 \u0644\u0628\u062f\u0621 \u0639\u0644\u0627\u062c \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0644\u062f\u0649 \u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a. <strong>\u062a\u062f\u0639\u0645 \u0627\u0644\u0623\u062f\u0644\u0629 \u0627\u0644\u0645\u0633\u062a\u0645\u062f\u0629 \u0645\u0646 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0645\u0627 \u0642\u0628\u0644 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u0645\u0639\u0631\u0648\u0636\u0629 \u0647\u0646\u0627 \u0641\u0643\u0631\u0629 \u0628\u062f\u0621 \u0627\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0641\u064a \u0633\u0646 \u0645\u0628\u0643\u0631\u0629\u060c \u0642\u0628\u0644 \u062d\u062f\u0648\u062b \u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0623\u0646\u0633\u062c\u0629 \u0627\u0644\u0645\u062a\u0642\u062f\u0645\u0629.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u062a\u0627\u0628\u0639 \u0627\u0644\u0642\u0631\u0627\u0621\u0629:<\/strong> <a href=\"https:\/\/www.biorxiv.org\/content\/10.64898\/2025.12.23.696144v1.full.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">\u064a\u064f\u0639\u062f \u0627\u0644\u062a\u0648\u0642\u064a\u062a \u0639\u0627\u0645\u0644\u0627\u064b \u0645\u0647\u0645\u0627\u064b: \u064a\u0643\u0648\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0623\u0643\u062b\u0631 \u0641\u0639\u0627\u0644\u064a\u0629 \u0639\u0646\u062f \u0627\u0644\u0628\u062f\u0621 \u0628\u0647 \u0645\u0628\u0643\u0631\u0627\u064b \u0641\u064a \u0646\u0645\u0648\u0630\u062c \u0641\u0623\u0631 \u0645\u0635\u0627\u0628 \u0628\u0636\u0645\u0648\u0631 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), aprogressive muscle wasting disorder caused by pathogenic variants in the DMD gene that typically disrupt the translation reading frame. This approach aims to modulate DMD pre-mRNA splicing to reframe the transcript and generate an internally deleted but partially functional quasi-dystrophin protein. >> Learn [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5418,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[473,119,77,178,61,472,268,471],"class_list":["post-5404","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-casimersen","tag-eteplirsen","tag-exon-skipping","tag-exon-skipping-therapies","tag-gene-therapy","tag-golodiresen","tag-microrna","tag-viltolarsen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/5404","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=5404"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/5404\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/5418"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=5404"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=5404"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=5404"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}