{"id":4512,"date":"2025-09-19T13:20:49","date_gmt":"2025-09-19T10:20:49","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4512"},"modified":"2025-09-19T17:03:29","modified_gmt":"2025-09-19T14:03:29","slug":"fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"FDA, Duchenne Musk\u00fcler Distrofi Tedavisi i\u00e7in NS-051\/NCNP-04&#039;e Yetim \u0130la\u00e7 Tan\u0131m\u0131 Verdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Nippon Shinyaku, ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nin (FDA), Duchenne Musk\u00fcler Distrofi (DMD) tedavisi i\u00e7in geli\u015ftirilmekte olan NS-051\/NCNP-04&#039;e Yetim \u0130la\u00e7 Tan\u0131m\u0131 verdi\u011fini duyurdu.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ABD&#039;de Yetim \u0130la\u00e7 Tan\u0131m\u0131, 200.000&#039;den az hastay\u0131 etkileyen hastal\u0131klar\u0131n tedavisine verilir ve bu tedavilerin geli\u015ftirilmesini ve de\u011ferlendirilmesini destekler. Yetim \u0130la\u00e7 Tan\u0131m\u0131, NS-051\/NCNP-04&#039;e yedi y\u0131ll\u0131k m\u00fcnhas\u0131r pazarlama haklar\u0131 sa\u011flar. <a href=\"https:\/\/dmdwarrior.com\/tr\/fda-grants-rare-pediatric-disease-designation-to-nippon-shinyakus-exon-51-skipping-study-ns-051-ncnp-04\/\" target=\"_blank\" rel=\"noreferrer noopener\">NS-051\/NCNP-04&#039;e Ocak 2025&#039;te FDA taraf\u0131ndan Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131 verildi.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DMD, distrofin proteininin eksikli\u011finden kaynaklanan ilerleyici bir kas erimesi hastal\u0131\u011f\u0131d\u0131r. \u0130skelet, kalp ve solunum kaslar\u0131nda g\u00fc\u00e7s\u00fczl\u00fc\u011fe yol a\u00e7ar. DMD&#039;ye neden olabilecek bir\u00e7ok genetik mutasyon t\u00fcr\u00fc vard\u0131r ve NS-051\/NCNP-04, ekzon 51 atlama tedavisine uygun, do\u011frulanm\u0131\u015f gen mutasyonu olan hastalar\u0131 tedavi etmek i\u00e7in geli\u015ftirilmektedir. Daha Fazla Bilgi Edinin: <a href=\"https:\/\/dmdwarrior.com\/tr\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ekzon 51 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">NS-051\/NCNP-04, Ulusal N\u00f6roloji ve Psikiyatri Merkezi (NCNP, Tokyo&#039;daki Kodaira \u015eehri; Ba\u015fkan, Kazuyuki Nakagome) ve Nippon Shinyaku taraf\u0131ndan ortak ke\u015ffedilen bir antisens oligon\u00fckleotiddir. NS-051\/NCNP-04, distrofin geninin genetik bilgisinin bir k\u0131sm\u0131n\u0131 atlar ve kas fonksiyonu bozulmas\u0131n\u0131 bask\u0131lama etkisine sahip olmas\u0131 beklenen, biraz daha k\u0131sa zincir uzunlu\u011funa sahip fonksiyonel bir distrofin proteini \u00fcretir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Nippon Shinyaku olarak, misyon bilinciyle, tedavisi zor ve nadir g\u00f6r\u00fclen hastal\u0131klar i\u00e7in yenilik\u00e7i yeni tedaviler geli\u015ftirmeye kendimizi adad\u0131k ve DMD hastalar\u0131na m\u00fcmk\u00fcn olan en k\u0131sa s\u00fcrede yeni tedavi se\u00e7enekleri sunmak i\u00e7in aktif olarak \u00e7al\u0131\u015f\u0131yoruz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/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>","protected":false},"excerpt":{"rendered":"<p>Nippon Shinyaku announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NS-051\/NCNP-04 which is being developed for the treatment of Duchenne Muscular Dystrophy (DMD). The Orphan Drug Designation in the U.S. is granted for treatments for diseases affecting fewer than 200,000 patients in the U.S., supporting their development and [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4514,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[122,193,421,194,136,192,195],"class_list":["post-4512","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-nippon-shinyaku","tag-ns-051-ncnp-04","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4512","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=4512"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4512\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4514"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4512"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4512"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4512"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}