{"id":4672,"date":"2025-09-29T16:14:48","date_gmt":"2025-09-29T13:14:48","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4672"},"modified":"2025-09-29T16:16:28","modified_gmt":"2025-09-29T13:16:28","slug":"dyne-therapeutics-receives-orphan-drug-designation-in-japan-for-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/dyne-therapeutics-receives-orphan-drug-designation-in-japan-for-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics, Duchenne Kas Distrofisi'nde DYNE-251 i\u00e7in Japonya'da Yetim \u0130la\u00e7 Tan\u0131m\u0131 Ald\u0131"},"content":{"rendered":"

Dyne Therapeutics, Japonya Sa\u011fl\u0131k, \u00c7al\u0131\u015fma ve Refah Bakanl\u0131\u011f\u0131'n\u0131n (MHLW), 51. ekson atlamaya uygun DMD gen mutasyonlar\u0131 olan Duchenne kas distrofisi (DMD) hastalar\u0131nda DYNE-251'ye Yetim \u0130la\u00e7 stat\u00fcs\u00fc verdi\u011fini duyurdu. DYNE-251, devam eden Faz 1\/2 DELIVER k\u00fcresel klinik \u00e7al\u0131\u015fmas\u0131nda de\u011ferlendiriliyor.<\/p>\n\n\n\n

\u00d6nemli Distrofin Ekspresyonu<\/h2>\n\n\n\n

Dyne'\u0131n ba\u015f t\u0131p g\u00f6revlisi Dr. Doug Kerr, "DELIVER deneyinde, \u00f6nemli distrofin ekspresyonuyla y\u00f6nlendirilen benzeri g\u00f6r\u00fclmemi\u015f ve s\u00fcrd\u00fcr\u00fclebilir i\u015flevsel iyile\u015fme g\u00f6rd\u00fck. Bu, DYNE-251'nin Duchenne kas distrofisiyle ya\u015fayan ki\u015filerin bak\u0131m\u0131n\u0131 d\u00f6n\u00fc\u015ft\u00fcrme potansiyelini vurguluyor" dedi. <\/strong>"ABD ve Avrupa'daki mevcut atamalar\u0131 tamamlayan yetim ila\u00e7 stat\u00fcs\u00fcn\u00fcn Japonya'da da verilmesiyle, DYNE-251, gelecek vaat eden bir yeni nesil ekzon 51 atlama tedavisi olarak ilerlemeye devam ediyor. Ekzon 51 atlamaya yatk\u0131n mutasyonlara sahip bireyler, DMD pop\u00fclasyonunda en yayg\u0131n ve i\u015flevsel olarak en \u00e7ok etkilenenler aras\u0131nda yer al\u0131yor. Bu kar\u015f\u0131lanmam\u0131\u015f ihtiyaca yan\u0131t verme potansiyeli, Duchenne toplulu\u011fu i\u00e7in i\u015flevsel iyile\u015fme sa\u011flayan bir tedavi geli\u015ftirme taahh\u00fcd\u00fcm\u00fcz\u00fc daha da art\u0131r\u0131yor ve Japonya'da DYNE-251 i\u00e7in d\u00fczenleyici yolu belirlemek \u00fczere Japon d\u00fczenleyicilerle i\u015f birli\u011fi yapmay\u0131 d\u00f6rt g\u00f6zle bekliyoruz."<\/p>\n\n\n\n

Japonya'da Yetim \u0130la\u00e7 DYNE-251 Olarak Belirlendi<\/h2>\n\n\n\n

Japonya'da Yetim \u0130la\u00e7 stat\u00fcs\u00fc, \u00fclkedeki 50.000'den az hastay\u0131 etkileyen ve y\u00fcksek t\u0131bbi ihtiya\u00e7 duyulan nadir hastal\u0131klar\u0131n tedavisi i\u00e7in tasarlanm\u0131\u015f ila\u00e7lara verilmektedir.<\/strong> Avantajlar aras\u0131nda geli\u015ftirme maliyetleri i\u00e7in s\u00fcbvansiyonlar ve onaylanmas\u0131 halinde 10 y\u0131la kadar potansiyel pazar ayr\u0131cal\u0131\u011f\u0131 yer almaktad\u0131r. DYNE-251 ayr\u0131ca, ABD G\u0131da ve \u0130la\u00e7 Dairesi'nden (FDA) \u00c7\u0131\u011f\u0131r A\u00e7an Tedavi unvan\u0131, H\u0131zl\u0131 Yol ve Nadir Pediatrik Hastal\u0131k unvanlar\u0131 ve FDA ile Avrupa \u0130la\u00e7 Ajans\u0131'ndan (EMA) DMD'li bireylerin tedavisi i\u00e7in ekson 51 atlamaya uygun Yetim \u0130la\u00e7 unvan\u0131 alm\u0131\u015ft\u0131r. Daha fazla bilgi edin<\/strong>: DYNE-251 i\u00e7in \u00c7\u0131\u011f\u0131r A\u00e7an Terapi Tan\u0131m\u0131<\/a><\/p>\n\n\n\n

O\u011flum ekzon 51 atlama terapilerine uygun mudur?<\/strong>: Ekzon 51 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n

\"Duchenne<\/figure>\n\n\n\n

DYNE-251 Hakk\u0131nda<\/h2>\n\n\n\n

DYNE-251, ekson 51 atlamaya uygun DMD gen mutasyonlar\u0131 olan DMD'li bireyler i\u00e7in Faz 1\/2 k\u00fcresel DELIVER klinik \u00e7al\u0131\u015fmas\u0131nda de\u011ferlendirilen ara\u015ft\u0131rma a\u015famas\u0131ndaki bir tedavidir. DYNE-251, transferin resept\u00f6r\u00fc 1'e (TfR1) ba\u011flanan bir antijen ba\u011flay\u0131c\u0131 par\u00e7aya (Fab) konjuge edilmi\u015f bir fosforodiamidat morfolino oligomerinden (PMO) olu\u015fur. Kas ve merkezi sinir sisteminde (MSS) neredeyse tam uzunlukta distrofin \u00fcretimini sa\u011flayarak i\u015flevsel iyile\u015fme sa\u011flamak \u00fczere tasarlanm\u0131\u015ft\u0131r. DYNE-251, ekson 51 atlamaya uygun DMD'li bireylerin tedavisi i\u00e7in ABD G\u0131da ve \u0130la\u00e7 Dairesi'nden (FDA) \u00c7\u0131\u011f\u0131r A\u00e7an Tedavi, H\u0131zl\u0131 Yol ve Nadir Pediatrik Hastal\u0131k unvanlar\u0131n\u0131n yan\u0131 s\u0131ra FDA ve Avrupa \u0130la\u00e7 Ajans\u0131'ndan (EMA) Yetim \u0130la\u00e7 unvan\u0131n\u0131 alm\u0131\u015ft\u0131r.<\/p>\n\n\n\n

Dyne, DYNE-251'ye ek olarak bir DMD franchise'\u0131 kuruyor ve 53, 45 ve 44 dahil olmak \u00fczere di\u011fer ekzonlar\u0131 hedef alan klinik \u00f6ncesi programlara sahip.<\/p>\n\n\n\n

Daha fazla bilgi edin<\/strong>: Duchenne Tedavisinde Yakla\u015fan Exon 51 Atlama Terapileri<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Dyne Therapeutics announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. DYNE-251 is being evaluated in the ongoing Phase 1\/2 DELIVER global clinical trial. […]<\/p>\n","protected":false},"author":1,"featured_media":4673,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,122,193,421,194,142],"class_list":["post-4672","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-japan"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4672","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=4672"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4672\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4673"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4672"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4672"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4672"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}