{"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\/tr\/wave-life-sciences-announces-plans-for-exon-53-skipping-study-in-2025\/","title":{"rendered":"Wave Life Sciences, 2025&#039;te Ekson 53 Atlama \u00c7al\u0131\u015fmas\u0131 Planlar\u0131n\u0131 Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u015eirketin \u00e7\u00f6zmeye \u00e7al\u0131\u015ft\u0131\u011f\u0131 kar\u015f\u0131lanmam\u0131\u015f ba\u015fl\u0131ca t\u0131bbi ihtiya\u00e7lar aras\u0131nda Alfa-1 antitripsin eksikli\u011fi (AATD), Huntington hastal\u0131\u011f\u0131 (HD), obezite ve Duchenne kas distrofisi (DMD) yer al\u0131yor. Haber, \u015firketin 13 Ocak 2025&#039;te 43. Y\u0131ll\u0131k JP Morgan Sa\u011fl\u0131k Konferans\u0131&#039;ndaki sunumundan \u00f6nce duyuruldu.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Exon 53 Atlama \u00c7al\u0131\u015fmas\u0131 Planlar\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>WVE-N531, ekzon 53 atlamaya uygun DMD&#039;li erkek \u00e7ocuklar\u0131n tedavisi i\u00e7in endojen, fonksiyonel distrofin proteininin \u00fcretimini te\u015fvik etmek \u00fczere tasarlanm\u0131\u015f bir ekzon atlama oligon\u00fckleotididir.<\/li>\n\n\n\n<li>2024&#039;teki \u00f6nemli ba\u015far\u0131lar\n<ul class=\"wp-block-list\">\n<li>WVE-N531&#039;in 2. Faz FORWARD-53 \u00e7al\u0131\u015fmas\u0131n\u0131n ara sonu\u00e7lar\u0131, olduk\u00e7a tutarl\u0131, ortalama kas i\u00e7eri\u011fi ayarl\u0131 distrofin ekspresyonunun 9.0% (aral\u0131\u011f\u0131: 4.6-13.9%) oldu\u011funu, s\u0131n\u0131f\u0131n\u0131n en iyisi kas iletimini, iyile\u015fmi\u015f kas sa\u011fl\u0131\u011f\u0131n\u0131n birden fazla g\u00f6stergesini ve g\u00fcvenli ve iyi tolere edilen bir profili g\u00f6sterdi.<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>Beklenen yak\u0131n gelecekteki kilometre ta\u015flar\u0131:\n<ul class=\"wp-block-list\">\n<li>FORWARD-53 \u00e7al\u0131\u015fmas\u0131 devam etmektedir ve t\u00fcm hastalar \u00e7al\u0131\u015fman\u0131n planlanan uzatma b\u00f6l\u00fcm\u00fcnde WVE-N531&#039;in ayl\u0131k dozlar\u0131yla tedaviye devam etmeyi se\u00e7mi\u015ftir.<\/li>\n\n\n\n<li>Wave&#039;in 48 haftal\u0131k FORWARD-53 verilerini teslim etmesi ve d\u00fczenleyicilerden h\u0131zland\u0131r\u0131lm\u0131\u015f onaya giden yol hakk\u0131nda geri bildirim almas\u0131 bekleniyor.<\/li>\n<\/ul>\n<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Alfa-1 antitripsin eksikli\u011fi (AATD), Huntington hastal\u0131\u011f\u0131 (HD), obezite ve Duchenne kas distrofisi (DMD), \u015firketin \u00e7\u00f6zmeye \u00e7al\u0131\u015ft\u0131\u011f\u0131 kar\u015f\u0131lanmam\u0131\u015f b\u00fcy\u00fck t\u0131bbi ihtiya\u00e7lar aras\u0131ndad\u0131r. Haber, \u015firketin 13 Ocak 2025&#039;te 43. Y\u0131ll\u0131k JP Morgan Sa\u011fl\u0131k Konferans\u0131&#039;ndaki sunumundan \u00f6nce yay\u0131nland\u0131. Exon 53 Atlama \u00c7al\u0131\u015fmas\u0131 Planlar\u0131<\/p>","protected":false},"author":1,"featured_media":2420,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[66,155,154,153],"class_list":["post-2418","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duchenne-muscular-dystrophy","tag-exon-53","tag-exon-53-skipping","tag-wave-life-sciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2418","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=2418"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/2418\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/2420"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=2418"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=2418"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=2418"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}