{"id":3925,"date":"2025-06-15T12:00:44","date_gmt":"2025-06-15T09:00:44","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3925"},"modified":"2025-06-15T12:00:45","modified_gmt":"2025-06-15T09:00:45","slug":"sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/ar\/sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients\/","title":{"rendered":"\u0623\u0641\u0627\u062f\u062a \u0634\u0631\u0643\u0629 Sarepta \u0623\u0646 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Elevidys \u0644\u0645\u0631\u0636 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 \u0644\u0646 \u064a\u064f\u0633\u062a\u062e\u062f\u0645 \u0641\u064a \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"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u062a\u0644\u0642\u0649 \u062f\u0648\u0627\u0621 \u0625\u064a\u0644\u064a\u0641\u064a\u062f\u064a\u0633 (\u062f\u064a\u0644\u0627\u0646\u062f\u0633\u062a\u0631\u0648\u062c\u064a\u0646 \u0645\u0648\u0643\u0633\u064a\u0628\u0627\u0631\u0641\u0648\u0641\u064a\u0643-\u0631\u0648\u0643\u0644)\u060c \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u0648\u062d\u064a\u062f \u0627\u0644\u0645\u0639\u062a\u0645\u062f \u0644\u0645\u0631\u0636\u0649 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646\u060c \u062a\u062d\u062f\u064a\u062b\u064b\u0627 \u0628\u0634\u0623\u0646 \u0633\u0644\u0627\u0645\u062a\u0647 \u0645\u0646 \u0634\u0631\u0643\u0629 Sarepta Therapeutics\u060c \u0627\u0644\u0634\u0631\u0643\u0629 \u0627\u0644\u0631\u0627\u0626\u062f\u0629 \u0641\u064a \u0645\u062c\u0627\u0644 \u0627\u0644\u0637\u0628 \u0627\u0644\u062c\u064a\u0646\u064a \u0627\u0644\u062f\u0642\u064a\u0642 \u0644\u0644\u0623\u0645\u0631\u0627\u0636 \u0627\u0644\u0646\u0627\u062f\u0631\u0629. \u0643\u0645\u0627 \u0646\u0627\u0642\u0634\u062a \u0627\u0644\u0634\u0631\u0643\u0629 \u0627\u0644\u0625\u062c\u0631\u0627\u0621\u0627\u062a \u0627\u0644\u062a\u064a \u062a\u062a\u062e\u0630\u0647\u0627 \u0644\u062a\u062d\u0633\u064a\u0646 \u0645\u0633\u062a\u0648\u0649 \u0627\u0644\u0633\u0644\u0627\u0645\u0629 \u0644\u062f\u0649 \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. \u0648\u0642\u062f \u0627\u062a\u064f\u062e\u0630\u062a \u0647\u0630\u0647 \u0627\u0644\u0625\u062c\u0631\u0627\u0621\u0627\u062a \u0639\u0642\u0628 \u062d\u0627\u0644\u0629 \u062b\u0627\u0646\u064a\u0629 \u0645\u0648\u062b\u0642\u0629 \u0645\u0646 \u0641\u0634\u0644 \u0627\u0644\u0643\u0628\u062f \u0627\u0644\u062d\u0627\u062f (ALF) \u0623\u062f\u062a \u0625\u0644\u0649 \u0627\u0644\u0648\u0641\u0627\u0629. \u0648\u062d\u062a\u0649 \u0627\u0644\u0622\u0646\u060c \u0643\u0627\u0646\u062a \u062d\u0627\u0644\u062a\u0627 \u0641\u0634\u0644 \u0627\u0644\u0643\u0628\u062f \u0627\u0644\u062d\u0627\u062f \u0644\u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u063a\u064a\u0631 \u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a. \u0648\u062a\u062a\u0642\u062f\u0645 \u0634\u0631\u0643\u0629 \u0633\u0627\u0631\u064a\u0628\u062a\u0627 \u0628\u0623\u062d\u0631 \u0627\u0644\u062a\u0639\u0627\u0632\u064a \u0644\u0623\u0633\u0631 \u0627\u0644\u0636\u062d\u0627\u064a\u0627 \u0648\u0645\u0642\u062f\u0645\u064a \u0627\u0644\u0631\u0639\u0627\u064a\u0629.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0648\u0641\u064a \u0627\u0644\u0648\u0642\u062a \u0646\u0641\u0633\u0647\u060c \u0643\u0634\u0641\u062a \u0634\u0631\u0643\u0629 \u0631\u0648\u0634 \u0627\u0644\u064a\u0648\u0645 \u0623\u064a\u0636\u064b\u0627 \u0639\u0646 \u0625\u0631\u0634\u0627\u062f\u0627\u062a \u0627\u0644\u062c\u0631\u0639\u0629 \u0627\u0644\u0645\u0646\u0642\u062d\u0629 \u0644\u062f\u0648\u0627\u0621 ELEVIDYS (delandistrogene moxeparvovec) \u0648\u0627\u0644\u062a\u064a \u0633\u062a\u062f\u062e\u0644 \u062d\u064a\u0632 \u0627\u0644\u062a\u0646\u0641\u064a\u0630 \u0639\u0644\u0649 \u0627\u0644\u0641\u0648\u0631 \u0644\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0630\u064a\u0646 \u064a\u0639\u0627\u0646\u0648\u0646 \u0645\u0646 \u0636\u0645\u0648\u0631 \u0627\u0644\u0639\u0636\u0644\u0627\u062a \u062f\u0648\u0634\u064a\u0646 \u063a\u064a\u0631 \u0627\u0644\u0645\u062a\u062d\u0631\u0643 (DMD)\u060c \u0628\u063a\u0636 \u0627\u0644\u0646\u0638\u0631 \u0639\u0646 \u0627\u0644\u0639\u0645\u0631\u060c \u0641\u064a \u0643\u0644 \u0645\u0646 \u0627\u0644\u0633\u064a\u0627\u0642\u0627\u062a \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0648\u0627\u0644\u062a\u062c\u0627\u0631\u064a\u0629. <strong>\u0644\u0627 \u064a\u0646\u0628\u063a\u064a \u0628\u0639\u062f \u0627\u0644\u0622\u0646 \u0625\u0639\u0637\u0627\u0621 Elevidys \u0644\u0644\u0645\u0631\u0636\u0649 \u063a\u064a\u0631 \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0641\u064a \u0628\u064a\u0626\u0629 \u062a\u062c\u0627\u0631\u064a\u0629.<\/strong> \u0633\u064a\u062a\u0645 \u0625\u064a\u0642\u0627\u0641 \u062a\u0633\u062c\u064a\u0644 \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 \u0641\u064a \u0627\u0644\u062a\u062c\u0627\u0631\u0628 \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0648\u062a\u062d\u062f\u064a\u062f \u062c\u0631\u0639\u0627\u062a\u0647\u0645 \u0641\u0648\u0631\u064b\u0627 \u0645\u0627 \u0644\u0645 \u064a\u062a\u0636\u0645\u0646 \u0628\u0631\u0648\u062a\u0648\u0643\u0648\u0644 \u0627\u0644\u0628\u062d\u062b \u0627\u0633\u062a\u0631\u0627\u062a\u064a\u062c\u064a\u0627\u062a \u0625\u0636\u0627\u0641\u064a\u0629 \u0644\u062a\u062e\u0641\u064a\u0641 \u0627\u0644\u0645\u062e\u0627\u0637\u0631 (\u0645\u062b\u0644 \u0627\u0644\u0623\u062f\u0648\u064a\u0629 \u0627\u0644\u0645\u0639\u062f\u0644\u0629 \u0644\u0644\u0645\u0646\u0627\u0639\u0629). \u062a\u064f\u0634\u0627\u0631\u0643 \u0627\u0644\u0645\u0639\u0644\u0648\u0645\u0627\u062a \u0645\u0639 \u0627\u0644\u0623\u0637\u0628\u0627\u0621 \u0648\u0627\u0644\u0628\u0627\u062d\u062b\u064a\u0646 \u0648\u0627\u0644\u062c\u0647\u0627\u062a \u0627\u0644\u0635\u062d\u064a\u0629 \u0644\u062a\u0639\u062f\u064a\u0644 \u0631\u0639\u0627\u064a\u0629 \u0627\u0644\u0645\u0631\u0636\u0649 \u0639\u0644\u0649 \u0627\u0644\u0641\u0648\u0631.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u0623\u0639\u0644\u0646\u062a \u0634\u0631\u0643\u0629 \u0633\u0627\u0631\u064a\u0628\u062a\u0627 \u0639\u0646 \u062b\u0627\u0646\u064a \u062d\u0627\u0644\u0629 \u0648\u0641\u0627\u0629 \u0628\u0633\u0628\u0628 \u0641\u0634\u0644 \u0627\u0644\u0643\u0628\u062f \u0628\u0639\u062f \u062a\u0644\u0642\u064a\u0647\u0627 \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u064a\u0623\u062a\u064a \u0647\u0630\u0627 \u0627\u0644\u0642\u0631\u0627\u0631 \u0628\u0639\u062f \u062a\u0642\u064a\u064a\u0645 \u062f\u0642\u064a\u0642 \u0644\u062d\u0627\u0644\u062a\u064a\u0646 \u0645\u0646 \u062d\u0627\u0644\u0627\u062a \u0641\u0634\u0644 \u0627\u0644\u0643\u0628\u062f \u0627\u0644\u062d\u0627\u062f \u0627\u0644\u0645\u0645\u064a\u062a (ALF) \u0644\u062f\u0649 \u0645\u0631\u0636\u0649 \u063a\u064a\u0631 \u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a. \u0648\u064f\u062c\u062f \u0623\u0646 Elevidys \u0648\u063a\u064a\u0631\u0647 \u0645\u0646 \u0627\u0644\u0639\u0644\u0627\u062c\u0627\u062a \u0627\u0644\u062c\u064a\u0646\u064a\u0629 \u0627\u0644\u0645\u0639\u062a\u0645\u062f\u0629 \u0639\u0644\u0649 AAV \u062a\u0646\u0637\u0648\u064a \u0639\u0644\u0649 \u0645\u062e\u0627\u0637\u0631\u060c \u0645\u0645\u0627 \u0627\u0633\u062a\u062f\u0639\u0649 \u0625\u0639\u0627\u062f\u0629 \u062a\u0642\u064a\u064a\u0645 \u0645\u0644\u0641 \u0627\u0644\u0641\u0648\u0627\u0626\u062f \u0648\u0627\u0644\u0645\u062e\u0627\u0637\u0631 \u0628\u0627\u0639\u062a\u0628\u0627\u0631\u0647 \u063a\u064a\u0631 \u0645\u0648\u0627\u062a\u064d \u0644\u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u063a\u064a\u0631 \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a. - \u0627\u0642\u0631\u0623 \u0627\u0644\u0645\u0632\u064a\u062f: <a href=\"https:\/\/dmdwarrior.com\/ar\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u0633\u0627\u0631\u064a\u0628\u062a\u0627\u060c \u062a\u064f\u0628\u0644\u063a \u0639\u0646 \u0648\u0641\u0627\u0629 \u0635\u0628\u064a \u064a\u0628\u0644\u063a \u0645\u0646 \u0627\u0644\u0639\u0645\u0631 16 \u0639\u0627\u0645\u064b\u0627 \u0628\u0639\u062f \u062a\u0644\u0642\u064a \u0627\u0644\u0639\u0644\u0627\u062c \u0627\u0644\u062c\u064a\u0646\u064a Elevidys<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0644\u0627 \u062a\u0624\u062b\u0631 \u0642\u064a\u0648\u062f \u0627\u0644\u062c\u0631\u0639\u0627\u062a \u0627\u0644\u062c\u062f\u064a\u062f\u0629 \u0639\u0644\u0649 \u0639\u0644\u0627\u062c \u0645\u0631\u0636\u0649 \u062f\u0648\u0634\u064a\u0646 \u0627\u0644\u0645\u062a\u0646\u0642\u0644\u064a\u0646 \u0645\u0646 \u0623\u064a \u0639\u0645\u0631\u060c \u0648\u062a\u0638\u0644 \u0646\u0633\u0628\u0629 \u0627\u0644\u0641\u0627\u0626\u062f\u0629 \u0625\u0644\u0649 \u0627\u0644\u0645\u062e\u0627\u0637\u0631 \u0625\u064a\u062c\u0627\u0628\u064a\u0629 \u0641\u064a \u0641\u0626\u0629 \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u0645\u062a\u0646\u0642\u0644\u064a\u0646.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u062d\u062f\u062b\u062a \u062d\u0627\u0644\u062a\u0627 \u0641\u0634\u0644 \u0643\u0644\u0648\u064a \u062d\u0627\u062f \u0642\u0627\u062a\u0644\u062a\u0627\u0646 \u0644\u062f\u0649 \u0645\u0631\u0636\u0649 \u063a\u064a\u0631 \u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a\u060c \u0645\u0646 \u0623\u0635\u0644 \u062d\u0648\u0627\u0644\u064a 140 \u0645\u0631\u064a\u0636\u064b\u0627 \u063a\u064a\u0631 \u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0639\u0648\u0644\u062c\u0648\u0627 \u0628\u0640 Elevidys \u0639\u0627\u0644\u0645\u064a\u064b\u0627 \u062d\u062a\u0649 \u0627\u0644\u0622\u0646. \u0628\u0639\u062f \u0623\u0648\u0644 \u062d\u0627\u0644\u0629 \u0641\u0634\u0644 \u0643\u0644\u0648\u064a \u062d\u0627\u062f \u0642\u0627\u062a\u0644\u0629\u060c \u0637\u0644\u0628\u062a \u0627\u0644\u062c\u0647\u0627\u062a \u0627\u0644\u062a\u0646\u0638\u064a\u0645\u064a\u0629 \u0627\u0644\u0623\u0648\u0631\u0648\u0628\u064a\u0629 \u0645\u0646 \u0631\u0648\u0634 \u0648\u0633\u0627\u0631\u064a\u0628\u062a\u0627 \u062a\u0639\u0644\u064a\u0642\u064b\u0627 \u0633\u0631\u064a\u0631\u064a\u064b\u0627 \u0645\u0624\u0642\u062a\u064b\u0627 \u0639\u0644\u0649 \u062f\u0631\u0627\u0633\u0627\u062a Elevidys \u0631\u0642\u0645 104 (NCT06241950)\u060c \u0648302 (ENVOL\u060c NCT06128564)\u060c \u0648303 (\u062f\u0631\u0627\u0633\u0629 ENVISION \u0631\u0642\u0645 303\u060c NCT05310071). \u0644\u0627 \u062a\u0632\u0627\u0644 \u0647\u0630\u0647 \u0627\u0644\u062a\u0639\u0644\u064a\u0642\u0627\u062a \u0627\u0644\u0633\u0631\u064a\u0631\u064a\u0629 \u0627\u0644\u0645\u0624\u0642\u062a\u0629 \u0633\u0627\u0631\u064a\u0629. \u062e\u0627\u0631\u062c \u0623\u0648\u0631\u0648\u0628\u0627\u060c \u0633\u064a\u062a\u0645 \u0625\u064a\u0642\u0627\u0641 \u0627\u0644\u062c\u0631\u0639\u0627\u062a \u0645\u0624\u0642\u062a\u064b\u0627\u060c \u0627\u0639\u062a\u0628\u0627\u0631\u064b\u0627 \u0645\u0646 \u0627\u0644\u0622\u0646\u060c \u0644\u062a\u062c\u0631\u0628\u0629 ENVISION. \u0643\u0645\u0627 \u0633\u062a\u0633\u0631\u064a \u0642\u064a\u0648\u062f \u0627\u0644\u062c\u0631\u0639\u0627\u062a \u0639\u0644\u0649 \u062c\u0631\u0639\u0627\u062a \u0627\u0644\u0645\u0631\u0636\u0649 \u0627\u0644\u062a\u062c\u0627\u0631\u064a\u064a\u0646 \u063a\u064a\u0631 \u0627\u0644\u0642\u0627\u062f\u0631\u064a\u0646 \u0639\u0644\u0649 \u0627\u0644\u0645\u0634\u064a \u0645\u0633\u062a\u0642\u0628\u0644\u064b\u0627.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u064a\u062a\u0639\u0644\u0645 \u0623\u0643\u062b\u0631: <a href=\"https:\/\/dmdwarrior.com\/ar\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">\u0627\u0644\u0623\u0633\u0626\u0644\u0629 \u0627\u0644\u0634\u0627\u0626\u0639\u0629 \u062d\u0648\u0644 Elevidys<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, has received a safety update from Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases. The company also discussed the actions it is taking to improve the safety profile in non-ambulatory patients. Following a second documented instance of acute [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3926,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[121,33,61,203,65],"class_list":["post-3925","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3925","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=3925"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/posts\/3925\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media\/3926"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/media?parent=3925"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/categories?post=3925"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/ar\/wp-json\/wp\/v2\/tags?post=3925"}],"curies":[{"name":"\u0648\u0648\u0631\u062f\u0628\u0631\u064a\u0633","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}