{"id":3223,"date":"2025-03-17T10:57:18","date_gmt":"2025-03-17T07:57:18","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3223"},"modified":"2025-03-17T11:00:40","modified_gmt":"2025-03-17T08:00:40","slug":"roche-to-share-ongoing-work-on-elevidys-gene-therapy-at-muscular-dystrophy-association-mda-2025-conference","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/roche-to-share-ongoing-work-on-elevidys-gene-therapy-at-muscular-dystrophy-association-mda-2025-conference\/","title":{"rendered":"\u7f57\u6c0f\u5c06\u5728 2025 \u5e74\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a (MDA) \u4f1a\u8bae\u4e0a\u5206\u4eab\u6b63\u5728\u8fdb\u884c\u7684 Elevidys \u57fa\u56e0\u6cbb\u7597\u7814\u7a76"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u5728\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a (MDA) 2025 \u5e74\u5927\u4f1a\u4e0a\uff0c\u7f57\u6c0f\u5c06\u8ba8\u8bba\u4ed6\u4eec\u6b63\u5728\u8fdb\u884c\u7684 Elevidys \u57fa\u56e0\u6cbb\u7597\u7814\u7a76\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5173\u4e8e Elevidys&#039; Embark \u4e24\u5e74\u6570\u636e\u4ee5\u53ca Study 101\u3001102 \u548c Endeavor \u7684\u6c47\u603b\u5206\u6790\u7684\u6700\u65b0\u53e3\u5934\u7814\u7a76\u663e\u793a\uff0c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7537\u5b69\u7684\u8fd0\u52a8\u529f\u80fd\u5173\u952e\u6307\u6807\u51fa\u73b0\u4e86\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u548c\u7edf\u8ba1\u5b66\u610f\u4e49\u7684\u6539\u5584<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Elevidys \u6b63\u5728\u8fdb\u884c\u7684\u4e34\u5e8a\u7814\u7a76<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys\uff0c\u767b\u8239\uff08A\u015fama III\uff0cNCT05096221\uff09tedaviden iki y\u0131l sonra ve \u00c7al\u0131\u015fma 101&#039;in\uff08A\u015fama I\/II\uff0cNCT03375164\uff0cn=4\uff09\uff0c\u00c7al\u0131\u015fma 102&#039;nin\uff08A\u015fama II\uff0cNCT03769116\uff0cn=26\uff09 ve ENDEAVOR Kohort 1&#039;in (A\u015fama Ib, NCT04626674 , n=20) birle\u015ftirilmi\u015f analizinde tedaviden \u00fc\u00e7 y\u0131l sonra motor fonksiyonun temel \u00f6l\u00e7\u00fcmlerinde ististiksel olarak anlaml\u0131 ve klinik olarak anlaml\u0131 iyile\u015fmeler g\u00f6sterdi,\u4f0a\u4f0a\u8fd9\u662f harici kontrol group kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda\u3002\u9876\u90e8 Elevidys \u662f tedavinin\uff0ctedavi s\u0131ras\u0131nda dort ila sekiz ya\u015flar\u0131ndaki bireylerde hastal\u0131\u011f\u0131n ilerlemesinin uzun vadeli \u7a33\u5b9asyonu veya yava\u015flamas\u0131 ile sonu\u00e7land\u0131\u011f\u0131n\u0131 g\u00f6stermektedir\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0e\u5339\u914d\u826f\u597d\u7684\u5916\u90e8\u5bf9\u7167\u7ec4\u76f8\u6bd4\uff0c\u5728 EMBARK\uff08III \u671f\uff0cNCT05096221\uff09\u6cbb\u7597\u4e24\u5e74\u540e\uff0c\u4ee5\u53ca\u5728\u7814\u7a76 101\uff08I\/II \u671f\uff0cNCT03375164\uff0cn=4\uff09\u3001\u7814\u7a76 102\uff08II \u671f\uff0cNCT03769116\uff0cn=26\uff09\u548c ENDEAVOR Cohort 1\uff08Ib \u671f\uff0cNCT04626674\uff0cn=20\uff09\u7684\u6c47\u603b\u5206\u6790\u4e2d\uff0c\u6cbb\u7597\u4e09\u5e74\u540e\uff0cElevidys \u5728\u8fd0\u52a8\u529f\u80fd\u5173\u952e\u6307\u6807\u65b9\u9762\u8868\u73b0\u51fa\u7edf\u8ba1\u5b66\u4e0a\u663e\u8457\u4e14\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u7684\u6539\u5584\u3002\u603b\u4e4b\uff0c\u8fd9\u4e9b\u6570\u636e\u8868\u660e\uff0c\u5bf9\u4e8e\u6cbb\u7597\u65f6\u5e74\u9f84\u4e3a 4 \u81f3 8 \u5c81\u7684\u4e2a\u4f53\uff0cElevidys \u6cbb\u7597\u53ef\u957f\u671f\u7a33\u5b9a\u6216\u51cf\u7f13\u75c5\u60c5\u8fdb\u5c55\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><a href=\"https:\/\/dmdwarrior.com\/zh\/roche-announces-new-results-from-the-embark-study-of-elevidys-in-outpatients-with-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"2612\" rel=\"noreferrer noopener\">EMBARK \u8bd5\u9a8c\u7b2c\u4e8c\u5e74\u7684\u9876\u7ebf\u6570\u636e\u4e8e 2025 \u5e74 1 \u6708\u516c\u5e03\u3002<\/a> \u5728\u4e24\u5e74\u7684 EMBARK \u7814\u7a76\u4e2d\u6ca1\u6709\u89c2\u5bdf\u5230\u65b0\u7684\u5b89\u5168\u4fe1\u53f7\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3a\u4e86\u8fdb\u4e00\u6b65\u8bc4\u4f30 Elevidys \u5bf9\u75be\u75c5\u8fdb\u5c55\u7684\u5f71\u54cd\uff0c\u5728 EMBARK \u7b2c\u4e00\u90e8\u5206\u4e2d\uff0c\u901a\u8fc7\u78c1\u5171\u632f\u6210\u50cf (MRI) \u5bf9\u90e8\u5206\u4e2a\u4f53\u7684\u808c\u8089\u5065\u5eb7\u548c\u808c\u8089\u75c5\u7406\u53d8\u5316\u8fdb\u884c\u4e86\u8bc4\u4f30\u3002\u7b2c 52 \u5468\u7684\u7ed3\u679c\u663e\u793a\uff0c\u4e0e\u63a5\u53d7\u5b89\u6170\u5242\u6cbb\u7597\u7684\u60a3\u8005\u76f8\u6bd4\uff0c\u63a5\u53d7 Elevidys \u6cbb\u7597\u7684\u60a3\u8005\u7684\u75be\u75c5\u8fdb\u5c55\u8d8b\u4e8e\u7a33\u5b9a\u6216\u51cf\u7f13\u3002\u7b2c 104 \u5468\uff0c\u4e0e\u7b2c 52 \u5468\u7684\u5b89\u6170\u5242\u76f8\u6bd4\uff0c\u808c\u8089\u548c\u808c\u8089\u7fa4\u7684 MRI \u53d8\u5316\u4e0e\u57fa\u7ebf\u76f8\u6bd4\u4ecd\u7136\u666e\u904d\u6709\u5229\u4e8e Elevidys\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/clinical-trials-review-of-elevidys-gene-therapy-is-elevidys-cost-worthy\/\" target=\"_blank\" data-type=\"post\" data-id=\"3027\" rel=\"noreferrer noopener\">Elevidys \u57fa\u56e0\u7597\u6cd5\uff1a\u503c\u5f97\u82b1\u94b1\u5417\uff1f<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Elevidys \u8425\u9500\u6388\u6743<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys \u5df2\u83b7\u6279\u7528\u4e8e\u6cbb\u7597 4 \u5c81\u4ee5\u4e0a\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\uff0c\u65e0\u8bba\u5176\u662f\u5426\u80fd\u591f\u884c\u8d70\uff0c\u5747\u5728\u7f8e\u56fd\u3001\u963f\u62c9\u4f2f\u8054\u5408\u914b\u957f\u56fd (UAE)\u3001\u5361\u5854\u5c14\u3001\u79d1\u5a01\u7279\u3001\u5df4\u6797\u548c\u963f\u66fc\u83b7\u5f97\u6279\u51c6\u3002Elevidys \u8fd8\u5df2\u83b7\u6279\u7528\u4e8e\u6cbb\u7597 4 \u81f3 7 \u5c81\u7684\u53ef\u884c\u8d70\u60a3\u8005\u3002\u8be5\u836f\u7269\u8fd8\u5df2\u5411\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u548c\u65e5\u672c\u3001\u745e\u58eb\u3001\u65b0\u52a0\u5761\u3001\u9999\u6e2f\u548c\u6c99\u7279\u963f\u62c9\u4f2f\u7684\u76d1\u7ba1\u673a\u6784\u63d0\u4ea4\u4e86\u7533\u8bf7\u3002\u8fc4\u4eca\u4e3a\u6b62\uff0c\u5df2\u6709 600 \u591a\u540d\u60a3\u8005\u63a5\u53d7\u4e86 Elevidys \u6cbb\u7597\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/why-isnt-roche-applying-marketing-authorization-in-every-country-for-elevidys-gene-therapy\/\">\u4e3a\u4ec0\u4e48\u7f57\u6c0f\u6ca1\u6709\u5728\u6bcf\u4e2a\u56fd\u5bb6\u7533\u8bf7Elevidys\u57fa\u56e0\u7597\u6cd5\u7684\u4e0a\u5e02\u8bb8\u53ef\uff1f<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>At the Muscular Dystrophy Association (MDA) 2025 Conference, Roche will discuss their ongoing work on Elevidys Gene Therapy. Late-breaking oral on Elevidys\u2019 Embark two-year data and pooled analysis of Study 101, 102 and Endeavor, demonstrated clinically meaningful and statistically significant improvements across key measures of motor function in boys with Duchenne muscular dystrophy (DMD) Elevidys [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3224,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[33,61,297,203],"class_list":["post-3223","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevidys","tag-gene-therapy","tag-muscular-dystrophy-association","tag-roche"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3223","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=3223"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3223\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3224"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3223"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3223"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3223"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}