{"id":3758,"date":"2025-04-24T14:48:38","date_gmt":"2025-04-24T11:48:38","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3758"},"modified":"2025-04-24T14:57:12","modified_gmt":"2025-04-24T11:57:12","slug":"dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"\u062d\u0635\u0644 Dyne Therapeutics \u0639\u0644\u0649 \u062a\u0635\u0646\u064a\u0641 \u0627\u0644\u0648\u0643\u0627\u0644\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629 \u0644\u0644\u0623\u062f\u0648\u064a\u0629 (EMA) \u0643\u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645 \u0644\u0640 DYNE-251 \u0641\u064a \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u0623\u0639\u0644\u0646\u062a \u0627\u0644\u064a\u0648\u0645 \u0634\u0631\u0643\u0629 Dyne Therapeutics\u060c \u0648\u0647\u064a \u0634\u0631\u0643\u0629 \u0641\u064a \u0645\u0631\u062d\u0644\u0629 \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0645\u062a\u062e\u0635\u0635\u0629 \u0641\u064a \u062a\u0637\u0648\u064a\u0631 \u0639\u0644\u0627\u062c\u0627\u062a \u0641\u0639\u0651\u0627\u0644\u0629 \u0644\u0644\u0623\u0634\u062e\u0627\u0635 \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0623\u0645\u0631\u0627\u0636 \u0639\u0635\u0628\u064a\u0629 \u0639\u0636\u0644\u064a\u0629 \u0648\u0631\u0627\u062b\u064a\u0629\u060c \u0623\u0646 DYNE-251 \u0642\u062f \u0635\u0646\u0641\u062a\u0647 \u0627\u0644\u0645\u0641\u0648\u0636\u064a\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629 \u0643\u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645 \u0644\u0639\u0644\u0627\u062c \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD). \u0648\u064a\u062c\u0631\u064a \u0627\u062e\u062a\u0628\u0627\u0631 DYNE-251 \u0639\u0644\u0649 \u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0645\u0639\u0631\u0636\u064a\u0646 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51 \u0641\u064a \u0627\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u0639\u0627\u0644\u0645\u064a\u0629 DELIVER \u0644\u0644\u0645\u0631\u062d\u0644\u0629 1\/2.<a href=\"https:\/\/dmdwarrior.com\/ar\/exon-51-skipping-therapies-dmd\/\" data-type=\"post\" data-id=\"3429\">\u0639\u0644\u0627\u062c\u0627\u062a \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 51<\/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=\"#phase-1-2-trial-of-dyne-251\">\u062a\u062c\u0631\u0628\u0629 \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2 \u0645\u0646 DYNE-251<\/a><\/li><li><a href=\"#what-is-orphan-drug\">\u0645\u0627 \u0647\u0648 \u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u064a\u062a\u064a\u0645\u061f<\/a><\/li><li><a href=\"#key-milestones-for-the-deliver-trial\">\u0623\u0647\u0645 \u0627\u0644\u0645\u0639\u0627\u0644\u0645 \u0641\u064a \u062a\u062c\u0631\u0628\u0629 DELIVER<\/a><\/li><li><a href=\"#about-dyne-251\">\u062d\u0648\u0644 DYNE-251<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"phase-1-2-trial-of-dyne-251\">\u062a\u062c\u0631\u0628\u0629 \u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2 \u0645\u0646 DYNE-251<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u0641\u064a \u0627\u0644\u0645\u0624\u062a\u0645\u0631 \u0627\u0644\u0633\u0631\u064a\u0631\u064a \u0648\u0627\u0644\u0639\u0644\u0645\u064a \u0644\u062c\u0645\u0639\u064a\u0629 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a (MDA) \u0644\u0639\u0627\u0645 \u0662\u0660\u0662\u0665\u060c \u0627\u0644\u0630\u064a \u0639\u064f\u0642\u062f \u0641\u064a \u0645\u0627\u0631\u0633\u060c \u0639\u064f\u0631\u0636\u062a \u0628\u064a\u0627\u0646\u0627\u062a \u0633\u0631\u064a\u0631\u064a\u0629 \u0637\u0648\u064a\u0644\u0629 \u0627\u0644\u0645\u062f\u0649 \u0645\u0646 \u062f\u0631\u0627\u0633\u0629 DELIVER \u0627\u0644\u062c\u0627\u0631\u064a\u0629 \u0644\u0640 DYNE-251. \u0623\u0638\u0647\u0631\u062a \u0647\u0630\u0647 \u0627\u0644\u0628\u064a\u0627\u0646\u0627\u062a \u062a\u062d\u0633\u0646\u064b\u0627 \u0648\u0638\u064a\u0641\u064a\u064b\u0627 \u063a\u064a\u0631 \u0645\u0633\u0628\u0648\u0642 \u0648\u0645\u0633\u062a\u062f\u0627\u0645\u064b\u0627 \u0639\u0646\u062f \u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0645\u064f\u062e\u062a\u0627\u0631\u0629. \u064a\u064f\u0639\u062f \u0645\u0642\u064a\u0627\u0633 \u0633\u0631\u0639\u0629 \u0627\u0644\u0646\u0628\u0636\u0629 \u0669\u0665 (SV95C)\u060c \u0648\u0647\u0648 \u0646\u062a\u064a\u062c\u0629 \u0631\u0642\u0645\u064a\u0629 \u0645\u0648\u0636\u0648\u0639\u064a\u0629 \u0645\u064f\u0639\u062a\u0631\u0641 \u0628\u0647\u0627 \u0643\u0646\u0642\u0637\u0629 \u0646\u0647\u0627\u064a\u0629 \u0623\u0633\u0627\u0633\u064a\u0629 \u0644\u0644\u062f\u0631\u0627\u0633\u0627\u062a \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0644\u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0641\u064a \u0623\u0648\u0631\u0648\u0628\u0627\u060c \u0623\u062d\u062f \u0627\u0644\u062a\u0642\u064a\u064a\u0645\u0627\u062a \u0627\u0644\u0648\u0638\u064a\u0641\u064a\u0629 \u0627\u0644\u0645\u064f\u0633\u062a\u062e\u062f\u0645\u0629 \u0641\u064a \u062a\u062c\u0631\u0628\u0629 DELIVER. (\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\/\">\u0627\u0644\u0645\u0631\u062d\u0644\u0629 1\/2 \u0645\u0646 \u062a\u062c\u0631\u0628\u0629 DELIVER \u0644\u0640 DYNE-251<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-orphan-drug\">\u0645\u0627 \u0647\u0648 \u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u064a\u062a\u064a\u0645\u061f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u0645\u0646\u062d \u0627\u0644\u0645\u0641\u0648\u0636\u064a\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629 \u062a\u0635\u0646\u064a\u0641 &quot;\u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645&quot; \u0644\u0644\u0623\u062f\u0648\u064a\u0629 \u0648\u0627\u0644\u0645\u0633\u062a\u062d\u0636\u0631\u0627\u062a \u0627\u0644\u0628\u064a\u0648\u0644\u0648\u062c\u064a\u0629 \u0627\u0644\u0645\u062e\u0635\u0635\u0629 \u0644\u0639\u0644\u0627\u062c \u0623\u0648 \u062a\u0634\u062e\u064a\u0635 \u0623\u0648 \u0627\u0644\u0648\u0642\u0627\u064a\u0629 \u0645\u0646 \u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0646\u0627\u062f\u0631\u0629 \u0623\u0648 \u0627\u0644\u0645\u0647\u062f\u062f\u0629 \u0644\u0644\u062d\u064a\u0627\u0629 \u0623\u0648 \u0627\u0644\u0645\u064f\u0646\u0647\u0643\u0629 \u0628\u0634\u0643\u0644 \u0645\u0632\u0645\u0646\u060c \u0623\u0648 \u0627\u0644\u062d\u0627\u0644\u0627\u062a \u0627\u0644\u062a\u064a \u062a\u064f\u0635\u064a\u0628 \u0623\u0642\u0644 \u0645\u0646 \u062e\u0645\u0633\u0629 \u0645\u0646 \u0643\u0644 \u0639\u0634\u0631\u0629 \u0622\u0644\u0627\u0641 \u0634\u062e\u0635 \u0641\u064a \u0627\u0644\u0627\u062a\u062d\u0627\u062f \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a. \u064a\u0648\u0641\u0631 \u062a\u0635\u0646\u064a\u0641 &quot;\u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645&quot; \u0644\u0644\u0634\u0631\u0643\u0627\u062a \u0645\u0632\u0627\u064a\u0627 \u0645\u064f\u0639\u064a\u0646\u0629\u060c \u062a\u0634\u0645\u0644 \u0631\u0633\u0648\u0645\u064b\u0627 \u062a\u0646\u0638\u064a\u0645\u064a\u0629 \u0645\u064f\u062e\u0641\u0636\u0629\u060c \u0648\u0645\u0633\u0627\u0639\u062f\u0629 \u0641\u064a \u0627\u0644\u0628\u0631\u0648\u062a\u0648\u0643\u0648\u0644\u0627\u062a \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629\u060c \u0648\u0645\u0646\u062d\u064b\u0627 \u0628\u062d\u062b\u064a\u0629\u060c \u0648\u0625\u0645\u0643\u0627\u0646\u064a\u0629 \u0627\u0644\u062d\u0635\u0648\u0644 \u0639\u0644\u0649 \u062d\u0642 \u0627\u0644\u062d\u0635\u0631\u064a\u0629 \u0641\u064a \u0627\u0644\u0633\u0648\u0642 \u0641\u064a \u0627\u0644\u0627\u062a\u062d\u0627\u062f \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a \u0644\u0645\u062f\u0629 \u062a\u0635\u0644 \u0625\u0644\u0649 \u0639\u0634\u0631 \u0633\u0646\u0648\u0627\u062a \u0641\u064a \u062d\u0627\u0644 \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629. \u0643\u0645\u0627 \u0645\u064f\u0646\u062d \u0627\u0644\u062f\u0648\u0627\u0621 DYNE-251 \u062a\u0635\u0646\u064a\u0641 &quot;\u062f\u0648\u0627\u0621 \u064a\u062a\u064a\u0645&quot; \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 \u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0623\u0637\u0641\u0627\u0644 \u0627\u0644\u0646\u0627\u062f\u0631\u0629 \u0641\u064a \u0645\u0627\u0631\u0633 2023.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"key-milestones-for-the-deliver-trial\">\u0623\u0647\u0645 \u0627\u0644\u0645\u0639\u0627\u0644\u0645 \u0641\u064a \u062a\u062c\u0631\u0628\u0629 DELIVER<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u062a\u0633\u062a\u0645\u0631 \u0634\u0631\u0643\u0629 Dyne \u0641\u064a \u0645\u062a\u0627\u0628\u0639\u0629 \u0645\u0633\u0627\u0631\u0627\u062a \u0627\u0644\u0645\u0648\u0627\u0641\u0642\u0629 \u0627\u0644\u0633\u0631\u064a\u0639\u0629 \u0639\u0627\u0644\u0645\u064a\u064b\u0627 \u0639\u0644\u0649 DYNE-251 \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 \u0648\u0627\u0644\u0630\u064a\u0646 \u064a\u0645\u0643\u0646\u0647\u0645 \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51.<\/li>\n\n\n\n<li>\u0642\u0627\u0645\u062a \u0634\u0631\u0643\u0629 \u062f\u0627\u064a\u0646 \u0628\u062a\u0633\u062c\u064a\u0644 \u0643\u0627\u0645\u0644 \u0645\u062c\u0645\u0648\u0639\u0629 \u062a\u0648\u0633\u064a\u0639 \u0627\u0644\u062a\u0633\u062c\u064a\u0644 \u0627\u0644\u0645\u0643\u0648\u0646\u0629 \u0645\u0646 32 \u0645\u0631\u064a\u0636\u064b\u0627 \u0643\u062c\u0632\u0621 \u0645\u0646 \u062a\u062c\u0631\u0628\u0629 DELIVER. \u0648\u0645\u0646 \u0627\u0644\u0645\u0642\u0631\u0631 \u0646\u0634\u0631 \u0628\u064a\u0627\u0646\u0627\u062a \u0647\u0630\u0647 \u0627\u0644\u0645\u062c\u0645\u0648\u0639\u0629 \u0641\u064a \u0623\u0648\u0627\u062e\u0631 \u0639\u0627\u0645 2025.<\/li>\n\n\n\n<li>\u0648\u062a\u062a\u0648\u0642\u0639 \u0634\u0631\u0643\u0629 \u062f\u0627\u064a\u0646 \u062a\u0642\u062f\u064a\u0645 \u0637\u0644\u0628 \u062a\u0631\u062e\u064a\u0635 \u0627\u0644\u0645\u0646\u062a\u062c\u0627\u062a \u0627\u0644\u0628\u064a\u0648\u0644\u0648\u062c\u064a\u0629 (BLA) \u0644\u0644\u062d\u0635\u0648\u0644 \u0639\u0644\u0649 \u0645\u0648\u0627\u0641\u0642\u0629 \u0633\u0631\u064a\u0639\u0629 \u0645\u0646 \u0627\u0644\u0648\u0644\u0627\u064a\u0627\u062a \u0627\u0644\u0645\u062a\u062d\u062f\u0629 \u0641\u064a \u0623\u0648\u0627\u0626\u0644 \u0639\u0627\u0645 2026.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"about-dyne-251\">\u062d\u0648\u0644 DYNE-251<\/h2>\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.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u062a\u0645 \u0645\u0646\u062d DYNE-251 \u062a\u0635\u0646\u064a\u0641\u0627\u062a \u0627\u0644\u062f\u0648\u0627\u0621 \u0627\u0644\u064a\u062a\u064a\u0645 \u0648\u0627\u0644\u0633\u0631\u064a\u0639 \u0648\u0627\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0646\u0627\u062f\u0631\u0629 \u0644\u062f\u0649 \u0627\u0644\u0623\u0637\u0641\u0627\u0644 \u0645\u0646 \u0642\u0628\u0644 \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 \u0644\u0639\u0644\u0627\u062c \u0637\u0641\u0631\u0627\u062a DMD \u0627\u0644\u0642\u0627\u0628\u0644\u0629 \u0644\u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 51.<\/p>","protected":false},"excerpt":{"rendered":"<p>Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3760,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,91,213,122,193,194,195],"class_list":["post-3758","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-ema","tag-european-medicines-agency","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3758","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=3758"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3758\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3760"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3758"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3758"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3758"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}