{"id":2418,"date":"2025-01-14T11:43:29","date_gmt":"2025-01-14T08:43:29","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2418"},"modified":"2025-03-07T14:58:15","modified_gmt":"2025-03-07T11:58:15","slug":"wave-life-sciences-announces-plans-for-exon-53-skipping-study-in-2025","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/wave-life-sciences-announces-plans-for-exon-53-skipping-study-in-2025\/","title":{"rendered":"\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 Wave Life Sciences \u0639\u0646 \u062e\u0637\u0637\u0647\u0627 \u0644\u0625\u062c\u0631\u0627\u0621 \u062f\u0631\u0627\u0633\u0629 \u062d\u0648\u0644 \u062a\u062e\u0637\u064a Exon 53 \u0641\u064a \u0639\u0627\u0645 2025"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u064a\u0639\u062f \u0646\u0642\u0635 \u0623\u0644\u0641\u0627-1 \u0623\u0646\u062a\u064a\u062a\u0631\u064a\u0628\u0633\u064a\u0646 (AATD)\u060c \u0648\u0645\u0631\u0636 \u0647\u0646\u062a\u0646\u063a\u062a\u0648\u0646 (HD)\u060c \u0648\u0627\u0644\u0633\u0645\u0646\u0629\u060c \u0648\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 (DMD) \u0645\u0646 \u0628\u064a\u0646 \u0627\u0644\u0627\u062d\u062a\u064a\u0627\u062c\u0627\u062a \u0627\u0644\u0637\u0628\u064a\u0629 \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u063a\u064a\u0631 \u0627\u0644\u0645\u0644\u0628\u0627\u0629 \u0627\u0644\u062a\u064a \u062a\u0633\u0639\u0649 \u0627\u0644\u0634\u0631\u0643\u0629 \u0625\u0644\u0649 \u062d\u0644\u0647\u0627. \u062a\u0645 \u0625\u0635\u062f\u0627\u0631 \u0627\u0644\u0623\u062e\u0628\u0627\u0631 \u0642\u0628\u0644 \u0627\u0644\u0639\u0631\u0636 \u0627\u0644\u062a\u0642\u062f\u064a\u0645\u064a \u0644\u0644\u0634\u0631\u0643\u0629 \u0641\u064a 13 \u064a\u0646\u0627\u064a\u0631 2025\u060c \u0641\u064a \u0627\u0644\u0645\u0624\u062a\u0645\u0631 \u0627\u0644\u0633\u0646\u0648\u064a \u0627\u0644\u062b\u0627\u0644\u062b \u0648\u0627\u0644\u0623\u0631\u0628\u0639\u064a\u0646 \u0644\u0644\u0631\u0639\u0627\u064a\u0629 \u0627\u0644\u0635\u062d\u064a\u0629 \u0644\u0634\u0631\u0643\u0629 \u062c\u064a \u0628\u064a \u0645\u0648\u0631\u062c\u0627\u0646.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u062e\u0637\u0637 \u0644\u062a\u062e\u0637\u064a \u062f\u0631\u0627\u0633\u0629 \u0625\u0643\u0633\u0648\u0646 53<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>WVE-N531 \u0647\u0648 \u0646\u064a\u0648\u0643\u0644\u064a\u0648\u062a\u064a\u062f \u062a\u062e\u0637\u064a \u0627\u0644\u0625\u0643\u0633\u0648\u0646 \u0645\u0635\u0645\u0645 \u0644\u062a\u062d\u0641\u064a\u0632 \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 \u062f\u0627\u062e\u0644\u064a \u0644\u0639\u0644\u0627\u062c \u0627\u0644\u0623\u0648\u0644\u0627\u062f \u0627\u0644\u0645\u0635\u0627\u0628\u064a\u0646 \u0628\u0645\u0631\u0636 \u062f\u0648\u0634\u064a\u0646 \u0648\u0627\u0644\u0630\u064a\u0646 \u064a\u0645\u0643\u0646 \u0623\u0646 \u064a\u062a\u062e\u0637\u0649 \u0627\u0644\u0625\u0643\u0633\u0648\u0646 53.<\/li>\n\n\n\n<li>\u0627\u0644\u0625\u0646\u062c\u0627\u0632\u0627\u062a \u0627\u0644\u0631\u0626\u064a\u0633\u064a\u0629 \u0641\u064a \u0639\u0627\u0645 2024\n<ul class=\"wp-block-list\">\n<li>\u0623\u0638\u0647\u0631\u062a \u0627\u0644\u0646\u062a\u0627\u0626\u062c \u0627\u0644\u0645\u0624\u0642\u062a\u0629 \u0644\u0644\u062f\u0631\u0627\u0633\u0629 \u0627\u0644\u062b\u0627\u0646\u064a\u0629 FORWARD-53 \u0644\u0640 WVE-N531 \u062b\u0628\u0627\u062a\u064b\u0627 \u0643\u0628\u064a\u0631\u064b\u0627 \u0641\u064a \u0645\u062a\u0648\u0633\u0637 \u0627\u0644\u062a\u0639\u0628\u064a\u0631 \u0639\u0646 \u0627\u0644\u062f\u064a\u0633\u062a\u0631\u0648\u0641\u064a\u0646 \u0627\u0644\u0645\u0639\u062f\u0644 \u062d\u0633\u0628 \u0645\u062d\u062a\u0648\u0649 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0628\u0645\u0642\u062f\u0627\u0631 9.0% (\u0627\u0644\u0646\u0637\u0627\u0642: 4.6-13.9%)\u060c \u0648\u0623\u0641\u0636\u0644 \u062a\u0648\u0635\u064a\u0644 \u0644\u0644\u0639\u0636\u0644\u0627\u062a \u0641\u064a \u0641\u0626\u062a\u0647\u060c \u0648\u0645\u0624\u0634\u0631\u0627\u062a \u0645\u062a\u0639\u062f\u062f\u0629 \u0644\u0635\u062d\u0629 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u0627\u0644\u0645\u062d\u0633\u0646\u0629\u060c \u0648\u0645\u0644\u0641 \u0622\u0645\u0646 \u0648\u062c\u064a\u062f \u0627\u0644\u062a\u062d\u0645\u0644.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>\u0627\u0644\u0645\u0639\u0627\u0644\u0645 \u0627\u0644\u0645\u062a\u0648\u0642\u0639\u0629 \u0627\u0644\u0642\u0627\u062f\u0645\u0629:\n<ul class=\"wp-block-list\">\n<li>\u0648\u062a\u0633\u062a\u0645\u0631 \u062a\u062c\u0631\u0628\u0629 FORWARD-53 \u0648\u0642\u062f \u0627\u062e\u062a\u0627\u0631 \u062c\u0645\u064a\u0639 \u0627\u0644\u0645\u0631\u0636\u0649 \u0645\u0648\u0627\u0635\u0644\u0629 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\u0627\u0644\u0634\u0631\u0643\u0629 \u0641\u064a 13 \u064a\u0646\u0627\u064a\u0631 2025\u060c \u0641\u064a \u0627\u0644\u0645\u0624\u062a\u0645\u0631 \u0627\u0644\u0633\u0646\u0648\u064a \u0627\u0644\u062b\u0627\u0644\u062b \u0648\u0627\u0644\u0623\u0631\u0628\u0639\u064a\u0646 \u0644\u0644\u0631\u0639\u0627\u064a\u0629 \u0627\u0644\u0635\u062d\u064a\u0629 \u0644\u0634\u0631\u0643\u0629 \u062c\u064a \u0628\u064a \u0645\u0648\u0631\u062c\u0627\u0646. \u062e\u0637\u0637 \u0644\u062f\u0631\u0627\u0633\u0629 \u062a\u062e\u0637\u064a \u0625\u0643\u0633\u0648\u0646 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