{"id":8921,"date":"2026-11-06T15:18:24","date_gmt":"2026-11-06T12:18:24","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8921"},"modified":"2026-11-06T15:27:18","modified_gmt":"2026-11-06T12:27:18","slug":"satellos-basecamp-phase-2-mri-muscle-fat-fraction","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/satellos-basecamp-phase-2-mri-muscle-fat-fraction\/","title":{"rendered":"Satellos, Duchenne Klinik \u00c7al\u0131\u015fmas\u0131nda Paz\u0131 Kas Ya\u011f Oran\u0131nda Azalma ve Daha D\u00fc\u015f\u00fck CK Seviyeleri Bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Satellos Bioscience Inc., ABD G\u0131da ve \u0130la\u00e7 \u0130daresi'nden ( FDA) gelen geri bildirimler do\u011frultusunda, Duchenne kas distrofisi (DMD) hastal\u0131\u011f\u0131nda Forazapadin'i de\u011ferlendiren BASECAMP Faz 2 pediatrik klinik \u00e7al\u0131\u015fmas\u0131n\u0131n birincil etkinlik sonlan\u0131m noktas\u0131nda bir de\u011fi\u015fiklik yapt\u0131\u011f\u0131n\u0131 duyurdu.<\/strong> \u015eirket, dinamometrenin yerini alacak \u015fekilde, yeni birincil etkinlik de\u011ferlendirme \u00f6l\u00e7\u00fct\u00fc olarak MR ile \u00f6l\u00e7\u00fclen kas ya\u011f oran\u0131n\u0131 se\u00e7ti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu karar, \u015firketin DMD'li yeti\u015fkinlerde y\u00fcr\u00fctt\u00fc\u011f\u00fc Faz 2 TRAILHEAD \u00e7al\u0131\u015fmas\u0131ndan elde edilen cesaret verici bulgular\u0131n ard\u0131ndan geldi. \u00c7al\u0131\u015fmada, biseps braki kas\u0131n\u0131n manyetik rezonans g\u00f6r\u00fcnt\u00fclemesi (MRI) ile \u00f6l\u00e7\u00fclen kas ya\u011f oran\u0131nda bir azalma g\u00f6sterilmi\u015fti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>TRAILHEAD \u00e7al\u0131\u015fmas\u0131ndaki kat\u0131l\u0131mc\u0131lar, Forazapadin ile be\u015f ay\u0131 a\u015fk\u0131n s\u00fcren tedavi boyunca, MRI ile \u00f6l\u00e7\u00fclen *biceps brachii* (paz\u0131) kas\u0131 ya\u011f oran\u0131nda ortalama y\u00fczde 3,7 puanl\u0131k bir azalma sergiledi; bu oran ba\u015flang\u0131\u00e7taki y\u00fczde 49,7 seviyesinden 140. g\u00fcnde y\u00fczde 46,0 seviyesine geriledi. Bu bulgu, kas kompozisyonunda bir iyile\u015fmeye i\u015faret etmektedir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Satellos ayr\u0131ca TRAILHEAD \u00e7al\u0131\u015fmas\u0131nda toplam eforda art\u0131\u015f, kas g\u00fcc\u00fcnde istikrar ve kreatin kinaz (CK) seviyelerinde d\u00fc\u015f\u00fc\u015f bildirdi. G\u00fcvenlik ve tolere edilebilirlik profili, daha \u00f6nce bildirilen verilerle tutarl\u0131 kald\u0131. Bu bulgular, MRI ile \u00f6l\u00e7\u00fclen kas ya\u011f fraksiyonundaki azalman\u0131n yan\u0131 s\u0131ra ek klinik g\u00f6zlemler sa\u011flamaktad\u0131r.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">BASECAMP Faz 2 \u00c7al\u0131\u015fmas\u0131: Yeni Birincil Sonu\u00e7 Noktas\u0131<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">BASECAMP \u00e7al\u0131\u015fmas\u0131, 7, 8 veya 9 ya\u015flar\u0131ndaki, ayakta tedavi g\u00f6ren 53 Duchenne Musk\u00fcler Distrofisi (DMD) hastas\u0131 erkek \u00e7ocukta Forazapadin'i de\u011ferlendiren, k\u00fcresel, randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc bir Faz 2 klinik \u00e7al\u0131\u015fmas\u0131d\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00c7al\u0131\u015fma, ba\u015flang\u0131\u00e7ta 12 haftal\u0131k plasebo kontroll\u00fc bir tedavi d\u00f6nemini ve ard\u0131ndan t\u00fcm kat\u0131l\u0131mc\u0131lar\u0131n Forazapadin ald\u0131\u011f\u0131 36 haftal\u0131k aktif bir tedavi d\u00f6nemini i\u00e7ermektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA'dan gelen geri bildirimler do\u011frultusunda, birincil etkinlik de\u011ferlendirme \u00f6l\u00e7\u00fct\u00fc art\u0131k MRI kullan\u0131larak vastus lateralis kas\u0131ndaki ya\u011f oran\u0131ndaki de\u011fi\u015fiklikleri \u00f6l\u00e7ecektir. \u0130kincil ve ke\u015fifsel de\u011ferlendirme \u00f6l\u00e7\u00fctleri ise kas kalitesini, fonksiyonel sonu\u00e7lar\u0131 ve Satellos'un tescilli Rejeneratif \u0130ndeksi de dahil olmak \u00fczere kas rejenerasyonunun biyobelirte\u00e7lerini de\u011ferlendirecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ya\u011f oran\u0131, kas dokusu i\u00e7indeki ya\u011f oran\u0131n\u0131n objektif bir MRI \u00f6l\u00e7\u00fcm\u00fcd\u00fcr. Duchenne kas distrofisinde, sa\u011fl\u0131kl\u0131 kas giderek ya\u011f ile yer de\u011fi\u015ftirir; bu nedenle kas ya\u011f oran\u0131ndaki de\u011fi\u015fiklikler, hastal\u0131\u011f\u0131n ilerlemesi ve kas bile\u015fimi a\u00e7\u0131s\u0131ndan \u00f6nemli bir \u00f6l\u00e7\u00fctt\u00fcr.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Satellos, 12 haftal\u0131k plasebo kontroll\u00fc BASECAMP d\u00f6nemine ait ilk sonu\u00e7lar\u0131 2027'nin ilk \u00e7eyre\u011finde a\u00e7\u0131klamay\u0131 bekliyor.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Forazapadin, v\u00fccudun iskelet kaslar\u0131n\u0131 onarma ve yenileme yetene\u011fini art\u0131rmak i\u00e7in tasarlanm\u0131\u015f, ara\u015ft\u0131rma a\u015famas\u0131nda olan oral yolla al\u0131nan k\u00fc\u00e7\u00fck molek\u00fcll\u00fc bir AAK1 inhibit\u00f6r\u00fcd\u00fcr. Duchenne kas distrofisi i\u00e7in potansiyel bir distrofin ba\u011f\u0131ms\u0131z tedavi olarak geli\u015ftirilmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha Fazlas\u0131n\u0131 Ke\u015ffedin<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/clinical-trials\/map\/\" target=\"_blank\" rel=\"noopener\">Duchenne Klinik Ara\u015ft\u0131rma Yerleri Haritas\u0131<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Satellos Bioscience Inc. has announced a change to the primary efficacy endpoint of its BASECAMP Phase 2 pediatric clinical trial evaluating Forazapadin in Duchenne muscular dystrophy (DMD), following feedback from the U.S. Food and Drug Administration (FDA). The company has selected MRI-measured muscle fat fraction as the new primary efficacy endpoint, replacing dynamometry. The decision [&hellip;]<\/p>\n","protected":false},"author":10,"featured_media":8922,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[922,903,251,252,253,829],"class_list":["post-8921","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-basecamp","tag-forazapadin","tag-sat-3247","tag-satellos","tag-satellos-bioscience","tag-trailhead"],"subtitle":"Satellos, Duchenne kas distrofisi ara\u015ft\u0131rmalar\u0131nda cesaret verici bulgular bildirdi; bu bulgular aras\u0131nda MR ile \u00f6l\u00e7\u00fclen biseps kas ya\u011f oran\u0131nda azalma ve s\u00fcrekli olarak daha d\u00fc\u015f\u00fck kreatin kinaz (CK) seviyeleri yer al\u0131yor. Bu bulgular, BASECAMP Faz 2 \u00e7al\u0131\u015fmas\u0131n\u0131n yeni birincil sonlanma noktas\u0131n\u0131 \u015fekillendiriyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8921","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\/10"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8921"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8921\/revisions"}],"predecessor-version":[{"id":8924,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8921\/revisions\/8924"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8922"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8921"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8921"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8921"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}