{"id":3876,"date":"2025-05-28T15:54:48","date_gmt":"2025-05-28T12:54:48","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3876"},"modified":"2025-11-03T20:23:36","modified_gmt":"2025-11-03T17:23:36","slug":"entrada-therapeutics-receives-eu-authorization-to-start-exon-45-skipping-clinical-trial-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/entrada-therapeutics-receives-eu-authorization-to-start-exon-45-skipping-clinical-trial-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Entrada Therapeutics, Duchenne Musk\u00fcler Distrofi'de Ekzon 45 Atlama Klinik Denemesine Ba\u015flamak \u0130\u00e7in AB Onay\u0131 Ald\u0131"},"content":{"rendered":"

Entrada Therapeutics, bug\u00fcn, Avrupa Birli\u011fi Klinik Ara\u015ft\u0131rma Y\u00f6netmeli\u011fi (EU-CTR) kapsam\u0131nda birden fazla \u00fclkenin Sa\u011fl\u0131k Otoriteleri ve Etik Komitelerinden, ekzon 45 atlamaya uygun Duchenne kas distrofisi (DMD) hastalar\u0131nda ENTR-601-45'nin Faz 1\/2 \u00e7oklu artan doz (MAD) klinik \u00e7al\u0131\u015fmas\u0131 olan ELEVATE-45-201'i ba\u015flatmak i\u00e7in yetki ald\u0131\u011f\u0131n\u0131 duyurdu.<\/p>\n\n\n\n

Entrada Therapeutics AB Onay\u0131 Ald\u0131<\/h2>\n\n\n\n

\u201cELEVATE-45-201, ekzon 45 atlamas\u0131na uygun bireyler i\u00e7in konjuge ekzon atlama terapisinin en geli\u015fmi\u015f klinik \u00e7al\u0131\u015fmas\u0131d\u0131r ve \u00f6nemli kar\u015f\u0131lanmam\u0131\u015f t\u0131bbi ihtiya\u00e7lar\u0131 olan b\u00fcy\u00fck bir Duchenne alt pop\u00fclasyonuna hitap etme potansiyeli sunmaktad\u0131r,\u201d dedi Entrada Therapeutics'nin \u0130cra Kurulu Ba\u015fkan\u0131 Dipal Doshi. \u201cAB onay\u0131, yeni Duchenne programlar\u0131m\u0131z\u0131n arkas\u0131ndaki b\u00fcy\u00fcyen klinik ivmeye dayanmaktad\u0131r. ELEVATE-44 ve ELEVATE-45'in yak\u0131n zamanda onaylanmas\u0131 ve bu y\u0131l\u0131n ilerleyen zamanlar\u0131nda ELEVATE-50 i\u00e7in planlanan ba\u015fvuru ile y\u0131l sonuna kadar DMD franchise'\u0131m\u0131z\u0131n kapsam\u0131n\u0131 \u00f6nemli \u00f6l\u00e7\u00fcde geni\u015fletme yolunday\u0131z.\u201d<\/p>\n\n\n\n

Devam\u0131n\u0131 oku:<\/strong> Duchenne Musk\u00fcler Distrofi Tedavisi \u0130\u00e7in Geli\u015ftirilen Yeni Nesil Ekzon Atlama Terapileri<\/a><\/p>\n\n\n\n

Ekzon 45 Atlama Klinik Deneyi AB'de Ba\u015fl\u0131yor<\/h2>\n\n\n\n

ELEVATE-45-201, ekzon 45 atlamaya uygun ayaktan Duchenne hastalar\u0131nda ENTR-601-45'nin g\u00fcvenli\u011fini, tolere edilebilirli\u011fini ve etkinli\u011fini de\u011ferlendiren k\u00fcresel, iki par\u00e7al\u0131, randomize, \u00e7ift k\u00f6r plasebo kontroll\u00fc bir Faz 1\/2 \u00e7al\u0131\u015fmas\u0131d\u0131r. B\u00f6l\u00fcm A, yakla\u015f\u0131k 24 hastada ekzon atlama ve distrofin \u00fcretimi dahil olmak \u00fczere g\u00fcvenli\u011fi, farmakokineti\u011fi ve farmakodinami\u011fini de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015f \u00e7oklu artan doz \u00e7al\u0131\u015fmas\u0131d\u0131r. Dozaj her alt\u0131 haftada bir uygulanacak ve \u00fc\u00e7 kohortta planlanan dozlar\u0131n 5 mg\/kg ile 15 mg\/kg aras\u0131nda de\u011fi\u015fmesi bekleniyor. \u00c7al\u0131\u015fman\u0131n B\u00f6l\u00fcm B'si, i\u015flevsel sonu\u00e7lar ve hasta taraf\u0131ndan bildirilen ya\u015fam kalitesi \u00f6l\u00e7\u00fcmleri dahil olmak \u00fczere g\u00fcvenlik ve etkinlik i\u00e7in B\u00f6l\u00fcm A'da belirlenen optimum dozu daha fazla de\u011ferlendirecektir. \u00c7al\u0131\u015fma kat\u0131l\u0131mc\u0131lar\u0131, ENTR-601-45'nin g\u00fcvenli\u011finin, etkinli\u011finin ve tolere edilebilirli\u011finin daha uzun bir s\u00fcre boyunca de\u011ferlendirilece\u011fi a\u00e7\u0131k etiketli bir uzatma \u00e7al\u0131\u015fmas\u0131na (OLE) kat\u0131lmaya uygun olabilir. \u015eirketin 2025'in 3. \u00e7eyre\u011finde ELEVATE-45-201'i ba\u015flatmas\u0131 bekleniyor.<\/p>\n\n\n\n

Devam\u0131n\u0131 oku<\/strong>:\u00a0Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Entrada Therapeutics, today announced it has received authorization from the Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial Regulation (EU-CTR) to initiate ELEVATE-45-201, a Phase 1\/2 multiple ascending dose (MAD) clinical study of ENTR-601-45 in patients living with Duchenne muscular dystrophy (DMD) who are amenable to exon 45 skipping. […]<\/p>\n","protected":false},"author":1,"featured_media":3881,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[320,91,321,234,317,318,319],"class_list":["post-3876","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevate-45-201","tag-ema","tag-entr-601-45","tag-entrada-therapeutics","tag-exon-45","tag-exon-45-skipping","tag-exon-45-skipping-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3876","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=3876"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3876\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3881"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3876"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3876"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3876"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}