{"id":4500,"date":"2025-09-18T14:58:30","date_gmt":"2025-09-18T11:58:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4500"},"modified":"2025-09-18T14:58:31","modified_gmt":"2025-09-18T11:58:31","slug":"del-zota-dmd-rna-therapy-from-avidity-biosciences-reverses-the-progression-of-disease","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/del-zota-dmd-rna-therapy-from-avidity-biosciences-reverses-the-progression-of-disease\/","title":{"rendered":"\u0639\u0644\u0627\u062c Del-Zota DMD RNA \u0645\u0646 Avidity Biosciences \u064a\u0639\u0643\u0633 \u062a\u0642\u062f\u0645 \u0627\u0644\u0645\u0631\u0636"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0628\u0633\u0628\u0628 \u0648\u0641\u0627\u0629 \u0645\u0631\u064a\u0636\u064a\u0646 \u0625\u062b\u0631 \u0627\u0633\u062a\u062e\u062f\u0627\u0645 Elevidys\u060c \u0648\u0647\u0648 \u0639\u0644\u0627\u062c \u062c\u064a\u0646\u064a \u0644\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a \u0645\u0646 Sarepta Therapeutics\u060c \u0645\u0631\u0651 \u0645\u062c\u062a\u0645\u0639 \u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u062f\u0648\u0634\u064a\u0646\u064a \u0628\u0639\u0627\u0645 \u0639\u0635\u064a\u0628. \u0648\u0642\u062f \u0635\u062f\u0645\u062a \u0647\u0630\u0647 \u0627\u0644\u062e\u0633\u0627\u0631\u0629 \u0639\u0627\u0626\u0644\u0627\u062a \u0627\u0644\u0645\u0631\u0636\u0649 \u0648\u0627\u0644\u0646\u0627\u0634\u0637\u064a\u0646 \u0648\u0627\u0644\u0628\u0627\u062d\u062b\u064a\u0646\u060c \u0645\u0645\u0627 \u0630\u0643\u0651\u0631\u0647\u0645 \u0628\u0645\u062e\u0627\u0637\u0631 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u062a\u062c\u0631\u064a\u0628\u064a \u0648\u0641\u0648\u0627\u0626\u062f\u0647 \u0627\u0644\u0645\u062d\u062a\u0645\u0644\u0629. <strong>\u0648\u0644\u0643\u0646 \u0641\u064a \u0648\u0642\u062a \u0633\u0627\u0628\u0642 \u0645\u0646 \u0647\u0630\u0627 \u0627\u0644\u0639\u0627\u0645\u060c \u0638\u0647\u0631 \u0628\u0635\u064a\u0635 \u0623\u0645\u0644 \u0648\u0633\u0637 \u0627\u0644\u062d\u0632\u0646 \u0648\u0639\u062f\u0645 \u0627\u0644\u064a\u0642\u064a\u0646: Del-Zota\u060c \u0648\u0647\u0648 \u0639\u0644\u0627\u062c RNA \u0645\u0646 Avidity Biosciences.<\/strong> \u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 \u0623\u0641\u064a\u062f\u064a\u062a\u064a \u0639\u0646 \u0646\u062c\u0627\u062d\u0647\u0627 \u0627\u0644\u0645\u064a\u0643\u0627\u0646\u064a\u0643\u064a \u0627\u0644\u0645\u0628\u0643\u0631 \u0641\u064a \u0627\u0644\u0631\u0628\u064a\u0639\u060c \u062d\u064a\u062b \u0627\u0633\u062a\u0639\u0627\u062f\u062a \u0625\u0646\u062a\u0627\u062c \u0628\u0631\u0648\u062a\u064a\u0646 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0634\u0628\u0647 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644 \u0628\u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0645\u0631\u062a\u0628\u0637\u0629 \u0628\u0627\u0644\u0645\u0631\u0636 \u063a\u064a\u0631 \u0627\u0644\u0645\u0635\u062d\u0648\u0628 \u0628\u0623\u0639\u0631\u0627\u0636\u060c \u0648\u0630\u0644\u0643 \u0628\u062a\u062c\u0627\u0648\u0632 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44 \u0641\u064a \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u0623\u0634\u0627\u0631\u062a \u0627\u0644\u0623\u062f\u0644\u0629 \u0622\u0646\u0630\u0627\u0643 \u0625\u0644\u0649 \u0623\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0642\u062f \u064a\u0643\u0648\u0646 \u0644\u0647 \u062a\u0623\u062b\u064a\u0631\u0627\u062a \u062c\u0632\u064a\u0626\u064a\u0629. \u0648\u0648\u0641\u0642\u064b\u0627 \u0644\u0623\u062d\u062f\u062b \u0627\u0644\u0642\u0631\u0627\u0621\u0627\u062a \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0644\u0634\u0631\u0643\u0629 \u0623\u0641\u064a\u062f\u064a\u062a\u064a \u0645\u0646 \u0628\u062d\u062b\u0647\u0627 \u0639\u0644\u0649 EXPLORE44\u060c \u0641\u0625\u0646 del-zota \u0644\u0627 \u064a\u0624\u062e\u0631 \u0641\u0642\u0637 \u0627\u0644\u062a\u0642\u062f\u0645 \u0627\u0644\u0645\u062a\u0648\u0627\u0635\u0644 \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0639\u0636\u0644\u064a\u060c \u0628\u0644 \u064a\u0628\u062f\u0648 \u0623\u0646\u0647 \u064a\u0639\u0643\u0633 \u0645\u0633\u0627\u0631\u0647.<\/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=\"#what-is-del-zota\">\u0645\u0627 \u0647\u0648 DEL-ZOTA\u061f<\/a><\/li><li><a href=\"#reverses-the-progression-of-disease\">\u064a\u0639\u0643\u0633 \u062a\u0642\u062f\u0645 \u0627\u0644\u0645\u0631\u0636<\/a><\/li><li><a href=\"#full-length-dystrophin-is-not-a-dream\">\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644 \u0644\u064a\u0633 \u062d\u0644\u0645\u0627<\/a><\/li><li><a href=\"#growing-and-building-muscle\">\u0646\u0645\u0648 \u0648\u0628\u0646\u0627\u0621 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0644\u062f\u0649 \u0627\u0644\u0623\u0648\u0644\u0627\u062f<\/a><\/li><li><a href=\"#a-patient-or-family-friendly-dosing-regimen\">\u0646\u0638\u0627\u0645 \u062c\u0631\u0639\u0627\u062a \u0645\u0646\u0627\u0633\u0628 \u0644\u0644\u0645\u0631\u064a\u0636 \u0623\u0648 \u0627\u0644\u0639\u0627\u0626\u0644\u0629<\/a><\/li><li><a href=\"#biologics-license-application-bla\">\u0637\u0644\u0628 \u062a\u0631\u062e\u064a\u0635 \u0627\u0644\u0645\u0648\u0627\u062f \u0627\u0644\u0628\u064a\u0648\u0644\u0648\u062c\u064a\u0629 (BLA)<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-del-zota\">\u0645\u0627 \u0647\u0648 DEL-ZOTA\u061f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u0645 \u062a\u0635\u0645\u064a\u0645 Del-zota \u0644\u062a\u0648\u0635\u064a\u0644 \u0623\u0648\u0644\u064a\u062c\u0648\u0645\u0631\u0627\u062a \u0641\u0648\u0633\u0641\u0648\u0631\u0648\u062f\u064a\u0627\u0645\u064a\u062f\u0627\u062a \u0627\u0644\u0645\u0648\u0631\u0641\u0648\u0644\u064a\u0646\u0648 (PMOs) \u0625\u0644\u0649 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0647\u064a\u0643\u0644\u064a\u0629 \u0648\u0623\u0646\u0633\u062c\u0629 \u0627\u0644\u0642\u0644\u0628 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44 \u0645\u0646 \u062c\u064a\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0628\u0634\u0643\u0644 \u062e\u0627\u0635 \u0644\u062a\u0645\u0643\u064a\u0646 \u0625\u0646\u062a\u0627\u062c \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0641\u064a \u0627\u0644\u0623\u0634\u062e\u0627\u0635 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u064a\u0634\u0648\u0646 \u0645\u0639 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 \u0645\u0639 \u0627\u0644\u0637\u0641\u0631\u0627\u062a \u0627\u0644\u0642\u0627\u0628\u0644\u0629 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44 (DMD44).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/ar\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\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 44 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"reverses-the-progression-of-disease\">\u064a\u0639\u0643\u0633 \u062a\u0642\u062f\u0645 \u0627\u0644\u0645\u0631\u0636<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">&quot;\u0644\u0642\u062f \u0634\u0647\u062f\u0646\u0627 \u0628\u0627\u0644\u0641\u0639\u0644 \u0627\u0646\u0639\u0643\u0627\u0633\u064b\u0627 \u0644\u0644\u0645\u0631\u0636&quot;\u060c \u0643\u0645\u0627 \u064a\u0642\u0648\u0644 \u0627\u0644\u062f\u0643\u062a\u0648\u0631 \u062f\u0628\u0644\u064a\u0648 \u0645\u0627\u064a\u0643\u0644 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646\u060c \u0643\u0628\u064a\u0631 \u0627\u0644\u0645\u0633\u0624\u0648\u0644\u064a\u0646 \u0627\u0644\u0639\u0644\u0645\u064a\u064a\u0646 \u0641\u064a \u0634\u0631\u0643\u0629 \u0623\u0641\u064a\u062f\u064a\u062a\u064a.<strong>\u0647\u0630\u0627 \u0644\u064a\u0633 \u0645\u062c\u0631\u062f \u0625\u0628\u0637\u0627\u0621 \u0644\u062a\u0637\u0648\u0631 \u0627\u0644\u0645\u0631\u0636\u060c \u0628\u0644 \u0647\u0648 \u0641\u064a \u0627\u0644\u0648\u0627\u0642\u0639 \u0627\u0646\u0639\u0643\u0627\u0633 \u0644\u0647. \u0648\u0647\u0630\u0647 \u0647\u064a \u0627\u0644\u0645\u0631\u0629 \u0627\u0644\u0623\u0648\u0644\u0649 \u0639\u0644\u0649 \u0627\u0644\u0625\u0637\u0644\u0627\u0642.<\/strong>\u2018&#039;\u2018<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0623\u0648\u0644\u064a\u063a\u0648\u0645\u0631 \u0641\u0648\u0633\u0641\u0648\u0646\u0648\u062f\u064a\u0627\u0645\u064a\u062f\u064a\u062a \u0645\u0648\u0631\u0641\u0648\u0644\u064a\u0646\u0648 (PMO)\u060c \u0648\u0647\u0648 \u0645\u0627\u062f\u0629 \u0643\u064a\u0645\u064a\u0627\u0626\u064a\u0629 \u0623\u062b\u0628\u062a\u062a \u0641\u0639\u0627\u0644\u064a\u062a\u0647\u0627 \u0641\u064a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646\u0627\u062a\u060c \u0647\u0648 \u062d\u0645\u0648\u0644\u0629 \u0627\u0644\u062d\u0645\u0636 \u0627\u0644\u0646\u0648\u0648\u064a \u0627\u0644\u0631\u064a\u0628\u0648\u0632\u064a (RNA) \u0627\u0644\u0645\u0641\u0636\u0644\u0629 \u0644\u062f\u0649 \u0623\u0641\u064a\u062f\u064a\u062a\u064a \u0644\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646. \u064a\u062a\u062c\u0627\u0648\u0632 \u0623\u0648\u0644\u064a\u063a\u0648\u0645\u0631 \u0641\u0648\u0633\u0641\u0648\u0646\u0648\u062f\u064a\u0627\u0645\u064a\u062f\u064a\u062a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0627\u0644\u062a\u0627\u0644\u0641 \u0641\u064a \u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0642\u0627\u0628\u0644 \u0644\u0644\u062a\u062d\u0648\u0644 \u0625\u0644\u0649 \u0625\u0643\u0633\u0648\u0646 44\u060c \u0648\u064a\u0633\u062a\u0639\u064a\u062f \u0625\u0637\u0627\u0631 \u0627\u0644\u0642\u0631\u0627\u0621\u0629\u060c \u0648\u064a\u0646\u062a\u062c \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0648\u0638\u064a\u0641\u064a\u064b\u0627.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"full-length-dystrophin-is-not-a-dream\">\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0643\u0627\u0645\u0644 \u0627\u0644\u0637\u0648\u0644 \u0644\u064a\u0633 \u062d\u0644\u0645\u0627<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0648\u0642\u0627\u0644 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646: &quot;\u0625\u0646\u0647\u0627 \u0641\u064a \u0627\u0644\u0648\u0627\u0642\u0639 \u0637\u0631\u064a\u0642\u0629 \u0644\u0644\u062a\u062e\u0644\u0635 \u0645\u0646 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0627\u0644\u062a\u0627\u0644\u0641 \u0648\u062a\u0648\u0641\u064a\u0631 \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0639\u0627\u0644\u064a \u0627\u0644\u062c\u0648\u062f\u0629 \u0628\u0637\u0648\u0644 \u0643\u0627\u0645\u0644 \u062a\u0642\u0631\u064a\u0628\u064b\u0627\u060c \u0648\u0647\u0630\u0627 \u0645\u0627 \u062a\u0645\u0643\u0646\u062a \u062a\u0642\u0646\u064a\u062a\u0646\u0627 \u0645\u0646 \u0641\u0639\u0644\u0647&quot;.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u0645 \u062a\u062c\u0646\u064a\u062f 26 \u0641\u062a\u0649 \u0648\u0634\u0627\u0628\u064b\u0627 \u064a\u062d\u0645\u0644\u0648\u0646 \u0637\u0641\u0631\u0627\u062a \u0641\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44 \u0644\u0644\u0628\u062d\u062b EXPLORE44 \u0645\u0646 \u0627\u0644\u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u0623\u0648\u0644\u0649 \/ \u0627\u0644\u062b\u0627\u0646\u064a\u0629\u060c \u0648\u0627\u0644\u0630\u064a \u0643\u0627\u0646 \u0639\u0634\u0648\u0627\u0626\u064a\u064b\u0627 \u0648\u062e\u0627\u0636\u0639\u064b\u0627 \u0644\u0644\u062a\u062d\u0643\u0645 \u0627\u0644\u0648\u0647\u0645\u064a \u0648\u0645\u0632\u062f\u0648\u062c \u0627\u0644\u062a\u0639\u0645\u064a\u0629. <strong>\u0648\u0643\u0627\u0646\u062a \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0623\u0648\u0644\u064a\u0629 \u0645\u0634\u062c\u0639\u0629: \u0625\u0630 \u0623\u062f\u0649 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44 \u0625\u0644\u0649 \u0627\u0646\u062e\u0641\u0627\u0636\u0627\u062a \u0643\u0628\u064a\u0631\u0629 \u0641\u064a \u0643\u064a\u0646\u0627\u0632 \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 (CK)\u060c \u0648\u0647\u0648 \u0645\u0624\u0634\u0631 \u062d\u064a\u0648\u064a \u0644\u0625\u0635\u0627\u0628\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0648\u0625\u0635\u0644\u0627\u062d \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0642\u0648\u064a.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/ar\/avidity-biosciences-del-zota-increase-dystrophin-phase-1-2-clinical-trial\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0623\u062b\u0628\u062a \u062f\u0648\u0627\u0621 \u062f\u064a\u0644-\u0632\u0648\u062a\u0627 (\u0639\u0644\u0627\u062c \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44) \u0642\u062f\u0631\u062a\u0647 \u0639\u0644\u0649 \u0639\u0643\u0633 \u0645\u0633\u0627\u0631 \u062a\u0642\u062f\u0645 \u0627\u0644\u0645\u0631\u0636 \u0639\u0628\u0631 \u0646\u0642\u0627\u0637 \u0627\u0644\u0646\u0647\u0627\u064a\u0629 \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0641\u064a \u062a\u062c\u0631\u0628\u0629 \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0641\u064a \u0627\u0644\u062d\u0627\u0644\u0627\u062a \u0627\u0644\u062a\u064a \u064a\u0648\u062c\u062f \u0641\u064a\u0647\u0627 \u0623\u0644\u064a\u0644 \u0648\u0627\u062d\u062f \u062a\u0627\u0644\u0641\u060c \u064a\u064f\u062d\u062a\u0645\u0644 \u0623\u0646 \u064a\u064f\u0646\u062a\u062c\u0648\u0627 \u062d\u0648\u0627\u0644\u064a 50% \u0648\u064a\u0638\u0644\u0648\u0646 \u0628\u062f\u0648\u0646 \u0623\u0639\u0631\u0627\u0636\u060c \u0643\u0645\u0627 \u0623\u0634\u0627\u0631 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646. &quot;\u0646\u0635\u0644 \u0625\u0644\u0649 \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0642\u0631\u064a\u0628\u0629 \u0645\u0646 \u0627\u0644\u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0627\u0644\u0637\u0628\u064a\u0639\u064a\u0629\u060c \u0623\u0648 \u0639\u0644\u0649 \u0627\u0644\u0623\u0642\u0644 \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u062e\u0627\u0644\u064a\u0629 \u0645\u0646 \u0627\u0644\u0623\u0639\u0631\u0627\u0636\u060c \u0645\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646. \u062a\u064f\u0638\u0647\u0631 \u0647\u0630\u0647 \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0623\u0646 \u0625\u0646\u0632\u064a\u0645 CK \u064a\u0646\u062e\u0641\u0636 \u0628\u0634\u0643\u0644 \u0643\u0628\u064a\u0631 \u0625\u0644\u0649 \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0642\u0631\u064a\u0628\u0629 \u0645\u0646 \u0627\u0644\u0637\u0628\u064a\u0639\u064a\u0629. \u0644\u0627 \u0623\u0639\u062a\u0642\u062f \u0623\u0646 \u0623\u064a \u0628\u0631\u0646\u0627\u0645\u062c \u0622\u062e\u0631 \u0641\u064a \u062f\u0648\u0634\u064a\u0646 \u0642\u062f \u0623\u0638\u0647\u0631 \u0639\u0648\u062f\u0629 \u0634\u0628\u0647 \u0637\u0628\u064a\u0639\u064a\u0629 \u0644\u0645\u0633\u062a\u0648\u064a\u0627\u062a CK. \u0648\u0646\u0631\u0649 \u0623\u0646 \u062d\u0648\u0627\u0644\u064a 50% \u0645\u0646 \u0627\u0644\u0623\u0634\u062e\u0627\u0635 \u0627\u0644\u0630\u064a\u0646 \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0640 del-zota \u0636\u0645\u0646 \u0627\u0644\u0646\u0637\u0627\u0642 \u0627\u0644\u0637\u0628\u064a\u0639\u064a. \u0623\u0645\u0627 \u0627\u0644\u0628\u0642\u064a\u0629\u060c \u0641\u0647\u0645 \u064a\u062d\u0648\u0645\u0648\u0646 \u062d\u0648\u0644 \u0627\u0644\u0646\u0637\u0627\u0642 \u0627\u0644\u0637\u0628\u064a\u0639\u064a.&quot; <a href=\"https:\/\/dmdwarrior.com\/ar\/what-is-creatine-kinase-ck\/\" data-type=\"post\" data-id=\"2167\" target=\"_blank\" rel=\"noreferrer noopener\">\u0645\u0627 \u0647\u0648 \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 \u0643\u064a\u0646\u0627\u0632\u061f<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"growing-and-building-muscle\">\u0646\u0645\u0648 \u0648\u0628\u0646\u0627\u0621 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0644\u062f\u0649 \u0627\u0644\u0623\u0648\u0644\u0627\u062f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0627\u0644\u062a\u063a\u064a\u064a\u0631\u0627\u062a \u0648\u0627\u0636\u062d\u0629 \u0644\u062f\u0649 \u0627\u0644\u0623\u0648\u0644\u0627\u062f \u0623\u0646\u0641\u0633\u0647\u0645: \u0635\u0639\u0648\u062f \u0627\u0644\u0633\u0644\u0627\u0644\u0645 \u0628\u0633\u0647\u0648\u0644\u0629 \u0623\u0643\u0628\u0631\u060c \u0648\u0627\u0644\u0648\u0642\u0648\u0641 \u0645\u0646 \u0627\u0644\u0623\u0631\u0636 \u0623\u0633\u0631\u0639\u060c \u0623\u0648 - \u0628\u064a\u0646 \u0627\u0644\u0645\u0631\u0636\u0649 \u063a\u064a\u0631 \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a - \u0627\u0644\u0648\u0635\u0648\u0644 \u0625\u0644\u0649 \u0645\u0633\u0627\u0641\u0629 \u0623\u0628\u0639\u062f \u0639\u0644\u0649 \u0637\u0627\u0648\u0644\u0629 \u0627\u0644\u0639\u0634\u0627\u0621. \u0642\u0627\u0644 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646: &quot;\u0645\u0627 \u0631\u0623\u064a\u0646\u0627\u0647 \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642. \u0647\u0630\u0627 \u0645\u0627 \u064a\u062d\u062f\u062b \u0639\u0646\u062f\u0645\u0627 \u062a\u064f\u0646\u062a\u062c \u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0639\u0627\u0644\u064a \u0627\u0644\u062c\u0648\u062f\u0629 \u0648\u062a\u062d\u0645\u064a \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0645\u0646 \u0627\u0644\u062a\u0644\u0641. \u064a\u0646\u0645\u0648 \u0627\u0644\u0623\u0648\u0644\u0627\u062f \u0648\u064a\u0628\u0646\u0648\u0646 \u0639\u0636\u0644\u0627\u062a\u0647\u0645\u060c \u0648\u0633\u062a\u0644\u0627\u062d\u0638\u0648\u0646 \u0647\u0630\u0627 \u0627\u0644\u0627\u0646\u0639\u0643\u0627\u0633 \u0641\u064a \u0645\u0633\u0627\u0631 \u0627\u0644\u0645\u0631\u0636 \u0639\u0628\u0631 \u0646\u0642\u0627\u0637 \u0646\u0647\u0627\u064a\u0629 \u0648\u0638\u064a\u0641\u064a\u0629 \u0645\u062a\u0639\u062f\u062f\u0629 \u0648\u0645\u062e\u062a\u0644\u0641\u0629. \u0647\u0630\u0627 \u062e\u0628\u0631 \u0633\u0627\u0631! \u0647\u0630\u0627 \u0645\u0627 \u064a\u0647\u0645 \u0627\u0644\u0646\u0627\u0633&quot;.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"a-patient-or-family-friendly-dosing-regimen\">\u0646\u0638\u0627\u0645 \u062c\u0631\u0639\u0627\u062a \u0645\u0646\u0627\u0633\u0628 \u0644\u0644\u0645\u0631\u064a\u0636 \u0623\u0648 \u0627\u0644\u0639\u0627\u0626\u0644\u0629<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u064a\u062a\u0645 \u062a\u062d\u062f\u064a\u062f \u062c\u0631\u0639\u0629 \u062f\u064a\u0644-\u0632\u0648\u062a\u0627 \u0643\u0644 \u0633\u062a\u0629 \u0623\u0633\u0627\u0628\u064a\u0639\u060c \u0648\u0647\u0648 \u0645\u0627 \u064a\u0637\u0644\u0642 \u0639\u0644\u064a\u0647 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646 &quot;\u0646\u0638\u0627\u0645 \u062c\u0631\u0639\u0627\u062a \u0645\u0646\u0627\u0633\u0628 \u0644\u0644\u0645\u0631\u064a\u0636 \u0623\u0648 \u0627\u0644\u0623\u0633\u0631\u0629&quot;\u060c \u0645\u0642\u0627\u0631\u0646\u0629 \u0628\u0628\u0639\u0636 \u0623\u062f\u0648\u064a\u0629 PMOs \u0627\u0644\u0645\u062a\u0648\u0641\u0631\u0629 \u0641\u064a \u0627\u0644\u0633\u0648\u0642\u060c \u0648\u0627\u0644\u062a\u064a \u062a\u062a\u0637\u0644\u0628 \u062c\u0631\u0639\u0627\u062a \u0623\u0633\u0628\u0648\u0639\u064a\u0629.<\/strong> \u0642\u0627\u0644 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646: &quot;\u0645\u0646 \u0627\u0644\u0635\u0639\u0628 \u0644\u0644\u063a\u0627\u064a\u0629 \u0639\u0644\u0649 \u0627\u0644\u0639\u0627\u0626\u0644\u0627\u062a \u0627\u0644\u0633\u0641\u0631 \u0648\u0627\u0644\u0645\u0634\u0627\u0631\u0643\u0629 \u0641\u064a \u0645\u062b\u0644 \u0647\u0630\u0647 \u0627\u0644\u0623\u0646\u0634\u0637\u0629\u060c \u0648\u0625\u0644\u0627 \u064a\u062c\u0628 \u0627\u0644\u0644\u062c\u0648\u0621 \u0625\u0644\u0649 \u0627\u0644\u062d\u0642\u0646 \u0627\u0644\u0645\u0646\u0632\u0644\u064a. \u0647\u0630\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0645\u0646\u0627\u0633\u0628 \u062c\u062f\u064b\u0627 \u0644\u0644\u0639\u0627\u0626\u0644\u0629. \u064a\u0645\u0643\u0646\u0643 \u0642\u0636\u0627\u0621 \u0639\u0637\u0644\u0629 \u0623\u0648 \u0631\u062d\u0644\u0629 \u0628\u062d\u0631\u064a\u0629 \u0648\u0627\u0644\u0645\u0634\u0627\u0631\u0643\u0629 \u0641\u064a \u0647\u0630\u0647 \u0627\u0644\u0623\u0646\u0634\u0637\u0629 \u0641\u064a \u0643\u0644 \u0645\u0631\u0629\u060c \u0645\u0639 \u062a\u0646\u0627\u0648\u0644 \u0627\u0644\u062c\u0631\u0639\u0629 \u0643\u0644 \u0633\u062a\u0629 \u0623\u0633\u0627\u0628\u064a\u0639. \u0646\u0639\u062a\u0642\u062f \u0623\u0646 \u0647\u0630\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0644\u064a\u0633 \u0641\u0642\u0637 \u0639\u0644\u0627\u062c\u064b\u0627 \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642 \u0644\u0644\u0645\u0631\u0636\u060c \u0628\u0644 \u064a\u062a\u0645\u064a\u0632 \u0623\u064a\u0636\u064b\u0627 \u0628\u062e\u0635\u0627\u0626\u0635 \u0623\u0645\u0627\u0646 \u0625\u064a\u062c\u0627\u0628\u064a\u0629\u060c \u0648\u0647\u0648 \u0645\u0646\u0627\u0633\u0628 \u062c\u062f\u064b\u0627 \u0644\u0644\u0645\u0631\u0636\u0649.&quot; <strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f<\/strong>: <a href=\"https:\/\/filecache.investorroom.com\/mr5ir_aviditybiosciences\/270\/Sep_10_2025_Del-zota_Fxnl_Data_Webcast_FINAL_09-09-25.pdf\" target=\"_blank\" rel=\"noopener\">\u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0644\u0640 Del-zota EXPLORE44-OLE<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"biologics-license-application-bla\">\u0637\u0644\u0628 \u062a\u0631\u062e\u064a\u0635 \u0627\u0644\u0645\u0648\u0627\u062f \u0627\u0644\u0628\u064a\u0648\u0644\u0648\u062c\u064a\u0629 (BLA)<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u062a\u0648\u0642\u0639 \u0634\u0631\u0643\u0629 \u0623\u0641\u064a\u062f\u064a\u062a\u064a \u062a\u0642\u062f\u064a\u0645 \u0623\u0648\u0644 \u0637\u0644\u0628 \u062a\u0631\u062e\u064a\u0635 \u0644\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0628\u064a\u0648\u0644\u0648\u062c\u064a\u0629 (BLA) \u0628\u062d\u0644\u0648\u0644 \u0646\u0647\u0627\u064a\u0629 \u0627\u0644\u0639\u0627\u0645 \u0628\u0645\u0648\u062c\u0628 \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0627\u0644\u0645\u064f\u0639\u062c\u0651\u0644\u0629. \u0648\u0635\u0631\u062d \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646: &quot;\u0646\u0639\u0645\u0644 \u0628\u062c\u062f\u0651 \u0639\u0644\u0649 \u0647\u0630\u0627 \u0627\u0644\u0623\u0645\u0631 \u062d\u0627\u0644\u064a\u064b\u0627\u060c \u0648\u0644\u062f\u064a\u0646\u0627 \u0623\u0642\u0633\u0627\u0645 \u0643\u062a\u0627\u0628\u0629 \u0648\u0623\u0642\u0633\u0627\u0645 \u0645\u0631\u0627\u062c\u0639\u0629&quot;. \u0648\u0623\u0636\u0627\u0641: &quot;\u0633\u062a\u0643\u0648\u0646 \u0647\u0630\u0647 \u0645\u0648\u0627\u0641\u0642\u0629 \u0645\u064f\u0639\u062c\u0651\u0644\u0629. \u0648\u0628\u0627\u0644\u062a\u0627\u0644\u064a\u060c \u0644\u062f\u064a\u0646\u0627 \u062c\u0645\u064a\u0639 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0630\u064a \u0646\u062d\u062a\u0627\u062c\u0647. \u0641\u064a \u0627\u0644\u0648\u0627\u0642\u0639\u060c \u0623\u0636\u0641\u0646\u0627 \u0645\u0631\u0636\u0649 \u0641\u064a \u0646\u0647\u0627\u064a\u0629 \u0627\u0644\u0639\u0627\u0645 \u0627\u0644\u0645\u0627\u0636\u064a \u0644\u0628\u0646\u0627\u0621 \u0642\u0627\u0639\u062f\u0629 \u0628\u064a\u0627\u0646\u0627\u062a \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0627\u0644\u062e\u0627\u0635\u0629 \u0628\u0646\u0627\u060c \u0644\u0643\u0646\u0646\u0627 \u0644\u0645 \u0646\u0643\u0646 \u0628\u062d\u0627\u062c\u0629 \u0625\u0644\u0649 \u0623\u064a \u0645\u0631\u0636\u0649 \u0625\u0636\u0627\u0641\u064a\u064a\u0646 \u0644\u0623\u062e\u0630 \u0627\u0644\u062e\u0632\u0639\u0627\u062a. \u0644\u0630\u0627\u060c \u0643\u0627\u0646 \u0630\u0644\u0643 \u0645\u064f\u0631\u064a\u062d\u064b\u0627 \u0644\u0644\u063a\u0627\u064a\u0629 \u0644\u0644\u0645\u0631\u0636\u0649\u060c \u062d\u064a\u062b \u0643\u0627\u0646 \u0644\u062f\u064a\u0646\u0627 \u0641\u0631\u0642 \u0643\u0627\u0641\u064d \u0641\u064a \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646\u060c \u0645\u0645\u0627 \u062c\u0639\u0644 \u0627\u0644\u0648\u0643\u0627\u0644\u0629 \u0645\u064f\u0642\u062a\u0646\u0639\u0629 \u0628\u0623\u0646 \u0645\u0633\u062a\u0648\u064a\u0627\u062a \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0644\u062f\u064a\u0646\u0627 \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642\u0629&quot;. \u0645\u0639 \u062d\u0635\u0648\u0644\u0646\u0627 \u0639\u0644\u0649 \u062a\u0635\u0646\u064a\u0641 \u0645\u064f\u0628\u062a\u0643\u0631\u060c \u064a\u0623\u0645\u0644 \u0641\u0644\u0627\u0646\u0627\u063a\u0627\u0646 \u0641\u064a \u0645\u0631\u0627\u062c\u0639\u0629 \u0633\u0631\u064a\u0639\u0629 \u0644\u0640 FDA\u060c \u0648\u0625\u0637\u0644\u0627\u0642 \u0645\u064f\u062d\u062a\u0645\u0644 \u0641\u064a \u0639\u0627\u0645\u064a 2026 \u0623\u0648 2027.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/ar\/upcoming-exon-44-skipping-therapies-for-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 44 \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<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Due to the deaths of two patients following the use of Elevidys, a DMD gene therapy from Sarepta Therapeutics, the DMD community has had a difficult year. Families, activists, and researchers were all shaken by the loss, which served as a sobering reminder of the dangers and potential benefits of experimental genetic therapy. But earlier [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4508,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[126,298,397,141,127,246,299],"class_list":["post-4500","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44"],"subtitle":"\u0623\u062f\u0649 \u0627\u0644\u0639\u0644\u0627\u062c \u0628\u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 44 \u0645\u0646 Del-Zota \u0625\u0644\u0649 \u0627\u0646\u062e\u0641\u0627\u0636\u0627\u062a \u0643\u0628\u064a\u0631\u0629 \u0641\u064a \u0643\u064a\u0646\u0627\u0632 \u0627\u0644\u0643\u0631\u064a\u0627\u062a\u064a\u0646 (CK) \u0648\u062a\u0644\u0641 \u0627\u0644\u0639\u0636\u0644\u0627\u062a\u060c \u0648\u0647\u0648 \u0645\u0624\u0634\u0631 \u062d\u064a\u0648\u064a \u0644\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD)\u060c \u0648\u0625\u0635\u0644\u0627\u062d \u0642\u0648\u064a \u0644\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/4500","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=4500"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/4500\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/4508"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=4500"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=4500"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=4500"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}