{"id":4399,"date":"2025-09-04T14:17:40","date_gmt":"2025-09-04T11:17:40","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4399"},"modified":"2025-09-04T14:30:44","modified_gmt":"2025-09-04T11:30:44","slug":"sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/sqy-therapeutics-orphan-drug-ema-exon-51-skipping-therapy-sqy51-for-duchenne\/","title":{"rendered":"SQY Therapeutics, Duchenne Musk\u00fcler Distrofi Tedavisi \u0130\u00e7in Ekzon 51 Atlama Terapisi SQY51 \u0130\u00e7in Avrupa \u0130la\u00e7 Ajans\u0131'ndan Yetim \u0130la\u00e7 Tan\u0131m\u0131 Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">SQY51, May\u0131s 2024&#039;te Duchenne Musk\u00fcler Distrofisi i\u00e7in ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nden Yetim \u0130la\u00e7 Tan\u0131m\u0131 (FDA) alm\u0131\u015ft\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SQY51, Duchenne Musk\u00fcler Distrofi&#039;den etkilenen erkek \u00e7ocuklar\u0131n\u0131n bir alt pop\u00fclasyonunda DMD geninin mRNA eklenmesi s\u0131ras\u0131nda ekzon-51&#039;i atlayarak yar\u0131 fonksiyonel distrofini geri kazand\u0131rmak \u00fczere tasarlanm\u0131\u015f bir palmitoil-konjuge tc-DNA antisens oligon\u00fckleotididir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/exon-51-skipping-therapies-dmd\/\">Duchenne Musk\u00fcler Distrofi Tedavisinde Yakla\u015fan Ekzon 51 Atlama Terapileri<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SQY Therapeutics \u00d6zel Bilimsel Dan\u0131\u015fman\u0131 Luis Garcia, &quot;FDA&#039;den ald\u0131\u011f\u0131m\u0131z ataman\u0131n ard\u0131ndan, EMA&#039;den de Yetim \u0130la\u00e7 Atamas\u0131 almaktan mutluluk duyuyoruz,&quot; dedi. &quot;Bu atamalar, Duchenne Musk\u00fcler Distrofi&#039;de kar\u015f\u0131lanmam\u0131\u015f \u00f6nemli bir ihtiyac\u0131 yans\u0131t\u0131yor ve SQY51&#039;in bu y\u0131k\u0131c\u0131 hastal\u0131ktan etkilenen ve ekzon-51 atlanmas\u0131ndan endi\u015fe duyan hastalar ve aileler i\u00e7in anlaml\u0131 bir fark yaratma potansiyeline olan inanc\u0131m\u0131z\u0131 peki\u015ftiriyor.&quot; <strong>SQY51<\/strong>: <a href=\"https:\/\/clinicaltrials.eu\/inn\/sqy51\/\" target=\"_blank\" rel=\"noreferrer noopener\">Duchenne Kas Distrofisi \u0130\u00e7in Umut Vadeden Bir \u0130la\u00e7<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SQY51, ekzon-51 atlamaya uygun DMD&#039;li bireylerde Faz 1\/2a AVANCE-1 klinik \u00e7al\u0131\u015fmas\u0131nda de\u011ferlendirilmektedir. \u015eirket, Faz 1\/2a&#039;ya 12 hastan\u0131n kayd\u0131n\u0131 tamamlam\u0131\u015f olup, verilerin 2026&#039;da toplanmas\u0131 beklenmektedir. Faz 1 tamamlanm\u0131\u015f olup Faz 2a \u00e7al\u0131\u015fmalar\u0131 devam etmektedir. SQY Therapeutics ayr\u0131ca, tedaviden faydalanmaya devam etmek isteyen kat\u0131l\u0131mc\u0131lar i\u00e7in bir uzatma faz\u0131 planlamaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">Duchenne Musk\u00fcler Distrofi Tedavisi \u0130\u00e7in Geli\u015ftirilen Yeni Nesil Ekzon Atlama Terapileri<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>SQY51 had already obtained Orphan Drug Designation from the US Food and Drug Administration (FDA) in Duchenne Muscular Dystrophy in May 2024. SQY51 is a palmitoyl-conjugated tc-DNA antisense oligonucleotide designed to restore semi-functional dystrophin by skipping exon-51 during mRNA splicing of the DMD gene in a subpopulation of boys affected by Duchenne Muscular Dystrophy. Read [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4403,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[91,213,122,193,194,410,195,409,408],"class_list":["post-4399","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ema","tag-european-medicines-agency","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-france","tag-orphan-drug","tag-sqy-therapeutics","tag-sqy51"],"subtitle":"SQY Therapeutics, Avrupa \u0130la\u00e7 Ajans\u0131'n\u0131n (EMA), Duchenne Musk\u00fcler Distrofisi hastalar\u0131n\u0131n tedavisi i\u00e7in distrofin pre-mRNA's\u0131n\u0131n ekzon-51'i hedef alan intraven\u00f6z bir molek\u00fcl olan SQY51'e Yetim \u0130la\u00e7 Tan\u0131m\u0131 (ODD) verdi\u011fini duyurdu.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4399","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=4399"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4399\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4403"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4399"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4399"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4399"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}