{"id":5484,"date":"2026-01-26T16:38:21","date_gmt":"2026-01-26T13:38:21","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5484"},"modified":"2026-01-26T23:15:59","modified_gmt":"2026-01-26T20:15:59","slug":"sarepta-3-year-topline-data-of-elevidys-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/sarepta-3-year-topline-data-of-elevidys-gene-therapy\/","title":{"rendered":"Sarepta, Duchenne Kas Distrofisi Olan Ayakta Tedavi G\u00f6ren Bireylerde ELEVIDYS Gen Terapisi ile \u0130lgili 3 Y\u0131ll\u0131k Verileri A\u00e7\u0131klad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Sarepta, tedavi s\u0131ras\u0131nda d\u00f6rt ila yedi ya\u015flar\u0131 aras\u0131nda olan ve ayakta tedavi g\u00f6ren Duchenne kas distrofisi hastalar\u0131nda ELEVIDYS&#039;i (delandistrogene moxeparvovec-rokl) de\u011ferlendiren, k\u00fcresel, randomize, plasebo kontroll\u00fc Faz 3 \u00e7al\u0131\u015fmas\u0131 EMBARK&#039;\u0131n (\u00c7al\u0131\u015fma 9001-301) Birinci B\u00f6l\u00fcm\u00fcnde tedavi edilen hastalardan elde edilen 3 y\u0131ll\u0131k fonksiyonel sonu\u00e7lar\u0131 a\u00e7\u0131klad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Sarepta, Duchenne kas distrofisinin en \u00f6nemli biyobelirteci olan kreatin kinaz d\u00fczeylerini 3 y\u0131ll\u0131k veriler aras\u0131nda a\u00e7\u0131klamad\u0131.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>ELEVIDYS ile tedavi edilen hastalar\u0131n ortalama ya\u015f\u0131 9 iken, tedaviden \u00fc\u00e7 y\u0131l sonra ba\u015flang\u0131\u00e7 de\u011ferlerinin \u00fczerinde ortalama North Star Ambulatory Assessment (NSAA) puanlar\u0131na ula\u015ft\u0131lar (n=52).<\/li>\n\n\n\n<li>ELEVIDYS gen terapisi, &quot;aya\u011fa kalkma s\u00fcresi&quot; (TTR) ve 10 metrelik y\u00fcr\u00fcme\/ko\u015fma (10MWR) ile \u00f6l\u00e7\u00fclen, e\u011filim a\u011f\u0131rl\u0131kl\u0131 d\u0131\u015f kontrol grubuna g\u00f6re gerileme oran\u0131nda 70% veya daha fazla azalma g\u00f6stermi\u015ftir. ELEVIDYS ile tedavi edilen hastalarda zaman i\u00e7inde artan bir tedavi etkisi g\u00f6r\u00fclm\u00fc\u015f ve d\u0131\u015f kontrol grubuna k\u0131yasla fonksiyonel fark 2. ve 3. y\u0131llar aras\u0131nda \u00f6nemli \u00f6l\u00e7\u00fcde geni\u015flemi\u015ftir.<\/li>\n\n\n\n<li>Tedaviyle ilgili yeni bir g\u00fcvenlik sinyali g\u00f6zlemlenmedi; bu durum, ELEVIDYS ile ayakta tedavi g\u00f6ren hastalarda bug\u00fcne kadar g\u00f6zlemlenen y\u00f6netilebilir g\u00fcvenlik profiliyle tutarl\u0131d\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne&#039;de Kuzey Y\u0131ld\u0131z\u0131 Ayakta Tedavi De\u011ferlendirmesi (NSAA)<\/a><\/p>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"778\" height=\"118\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png\" alt=\"Sarepta, Duchenne Kas Distrofisi Olan Ayakta Tedavi G\u00f6ren Bireylerde ELEVIDYS Gen Terapisi ile \u0130lgili 3 Y\u0131ll\u0131k Verileri A\u00e7\u0131klad\u0131\" class=\"wp-image-5496\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png 778w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-300x46.png 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-768x116.png 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-18x3.png 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-150x23.png 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-696x106.png 696w\" sizes=\"auto, (max-width: 778px) 100vw, 778px\" \/><\/figure>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td><strong>Fonksiyonel Sonu\u00e7lar<\/strong><\/td><td><strong>LSM De\u011fi\u015fim Fark\u0131 ve EC Kar\u015f\u0131la\u015ft\u0131rmas\u0131<\/strong><\/td><td><strong>p-De\u011feri<\/strong><\/td><\/tr><tr><td>NSAA<\/td><td>+4,39 puan (iyile\u015fme)<\/td><td>p=0.0002<\/td><\/tr><tr><td>TTR<\/td><td>-6,05 saniye (iyile\u015fme)<\/td><td>p&lt;0.0001<\/td><\/tr><tr><td>10MWR<\/td><td>-2,70 saniye (iyile\u015fme)<\/td><td>p=0.0039<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">26 Ocak 2026&#039;da Sarepta hisseleri 22 Amerikan dolar\u0131ndan i\u015flem g\u00f6rmeye ba\u015flad\u0131 ve Elevidys gen terapisiyle ilgili \u00fc\u00e7 y\u0131ll\u0131k verilerin a\u00e7\u0131klanmas\u0131n\u0131n ard\u0131ndan 24,68 Amerikan dolar\u0131na y\u00fckseldi. Ancak saatler sonra d\u00fc\u015f\u00fc\u015fe ge\u00e7erek Do\u011fu Zaman Dilimi&#039;ne g\u00f6re 13:04 itibariyle 22,52 Amerikan dolar\u0131ndan i\u015flem g\u00f6rd\u00fc. Bu d\u00fc\u015f\u00fc\u015f, yat\u0131r\u0131mc\u0131lar\u0131n Elevidys gen terapisiyle ilgili \u00fc\u00e7 y\u0131ll\u0131k verilerden memnun kalmad\u0131\u011f\u0131n\u0131n bir i\u015fareti olarak yorumland\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/tr\/creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys Gen Terapisinde Kreatin Kinaz (CK), Distrofin D\u00fczeyi ve NSAA Skoru<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Sarepta announced 3-year topline functional results from patients treated in Part One of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment. Sarepta were not disclosed creatine kinase levels, the most major biomarker [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5485,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[66,33,179,65],"class_list":["post-5484","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duchenne-muscular-dystrophy","tag-elevidys","tag-elevidys-gene-therapy","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5484","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5484"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5484\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5485"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5484"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5484"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5484"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}