{"id":4776,"date":"2025-10-10T17:06:47","date_gmt":"2025-10-10T14:06:47","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4776"},"modified":"2025-10-10T17:17:25","modified_gmt":"2025-10-10T14:17:25","slug":"new-data-of-sat-3247-in-first-in-human-trial-of-adults-with-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/new-data-of-sat-3247-in-first-in-human-trial-of-adults-with-duchenne-muscular-dystrophy\/","title":{"rendered":"Satellos, Duchenne Kas Distrofisi Olan Yeti\u015fkinlerde \u0130lk \u0130nsan Deneylerinde SAT-3247&#039;nin G\u00fcvenli\u011fini, Tolerans\u0131n\u0131 ve \u0130\u015flevsel Etkisini Daha da G\u00f6steren Yeni Verileri Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Satellos Bioscience, Avusturya&#039;n\u0131n Viyana kentinde d\u00fczenlenen D\u00fcnya Kas Derne\u011fi&#039;nin 30. Y\u0131ll\u0131k Kongresi&#039;nde, Duchenne kas distrofisi (Duchenne veya DMD) olan yeti\u015fkinlerde (20-27 ya\u015f) SAT-3247&#039;nin tolere edilebilirli\u011fini ve ba\u015flang\u0131\u00e7 etkinli\u011fini daha da g\u00f6steren yeni verileri duyurdu.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#key-findings\">Temel Bulgular<\/a><\/li><li><a href=\"#increase-in-grip-strength\">Kavrama G\u00fcc\u00fcnde Art\u0131\u015f<\/a><\/li><li><a href=\"#regardless-specific-dystrophin-mutation\">Belirli Ekzon Mutasyonundan Ba\u011f\u0131ms\u0131z<\/a><\/li><li><a href=\"#future-clinical-trial-plans\">Gelecekteki Klinik Ara\u015ft\u0131rma Planlar\u0131<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"key-findings\">Temel Bulgular<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Duchenne kas distrofisi olan yeti\u015fkinler, SAT-3247 ile 28 g\u00fcnl\u00fck tedaviyi takiben do\u011fal seyri a\u015fan i\u015flevsel iyile\u015fmeler g\u00f6sterdi<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Kavrama g\u00fcc\u00fc 118.6% artt\u0131 ve \u00f6ng\u00f6r\u00fclen zorunlu vital kapasite 5.8% artt\u0131; oysa her ikisi de do\u011fal tarihte azald\u0131<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>SAT-3247, Faz 1 a\/b \u00e7al\u0131\u015fmas\u0131n\u0131n t\u00fcm b\u00f6l\u00fcmlerinde g\u00fcvenlik, tolere edilebilirlik ve beklenen farmakokineti\u011fi (FK) g\u00f6stermi\u015ftir<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>D\u00fcnya Kas Toplulu\u011fu&#039;nun 30. Y\u0131ll\u0131k Kongresi&#039;nde son dakika poster oturumunda sunulan yeni veriler<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Satellos&#039;nin kurucu orta\u011f\u0131 ve CEO&#039;su Frank Gleeson, &quot;Satellos&#039;nin Duchenne&#039;li yeti\u015fkinlerde 28 g\u00fcnl\u00fck klinik \u00e7al\u0131\u015fmas\u0131ndan elde edilen yeni ve g\u00fcncellenmi\u015f klinik sonu\u00e7lar\u0131, SAT-3247&#039;nin g\u00fcvenli ve klinik a\u00e7\u0131dan anlaml\u0131 bir tedavi olma potansiyelinin \u00f6nemli bir do\u011frulamas\u0131n\u0131 sa\u011fl\u0131yor,&quot; dedi. &quot;SAT-3247&#039;nin Duchenne toplulu\u011funa d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc potansiyelini g\u00f6sterme hedefiyle klinik \u00e7al\u0131\u015fmalar\u0131m\u0131z\u0131n bir sonraki a\u015famas\u0131na ge\u00e7mekten heyecan ve g\u00fcven duyuyoruz.&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Toplant\u0131da sunulan veriler, SAT-3247&#039;nin Faz 1a\/b \u00e7al\u0131\u015fmas\u0131 boyunca g\u00fcvenli ve iyi tolere edildi\u011fini ve istenen farmakokinetik (FK) profile sahip oldu\u011funu g\u00f6stermektedir. Sunumda ayr\u0131ca, erken ila\u00e7 etkisinin ke\u015fifsel \u00f6l\u00e7\u00fcmlerine ili\u015fkin yeni analizler de yer alm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"increase-in-grip-strength\">Kavrama G\u00fcc\u00fcnde Art\u0131\u015f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>28 g\u00fcnl\u00fck bir s\u00fcre boyunca SAT-3247 ile tedavi edilen bireylerde, bu ya\u015f grubunda Duchenne do\u011fal ge\u00e7mi\u015fine g\u00f6re \u00e7ok daha fazla kavrama g\u00fcc\u00fc art\u0131\u015f\u0131 g\u00f6r\u00fcld\u00fc.<\/strong> \u00d6zellikle, bask\u0131n elde maksimum kavrama g\u00fcc\u00fcnde 118,6% ortalama iyile\u015fme, bask\u0131n olmayan elde ise 97,9% ortalama iyile\u015fme g\u00f6zlemlendi; bu da kavrama g\u00fcc\u00fcn\u00fcn yakla\u015f\u0131k 2 kg&#039;dan yakla\u015f\u0131k 4 kg&#039;a iki kat\u0131na \u00e7\u0131kt\u0131\u011f\u0131n\u0131 g\u00f6steriyor. Bu iyile\u015fmeler, yay\u0131nlanm\u0131\u015f do\u011fal seyirle uyu\u015fmuyor ve 15. g\u00fcndeki daha y\u00fcksek ila\u00e7 konsantrasyonlar\u0131 ve daha y\u00fcksek bazal kreatinin (artm\u0131\u015f kas k\u00fctlesinin bir g\u00f6stergesi) ile ili\u015fkiliydi; bunun, ilac\u0131m\u0131z\u0131n kas \u00fczerinde istenen etkiyi g\u00f6sterdi\u011fine inan\u0131yoruz.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ayr\u0131ca, kat\u0131l\u0131mc\u0131lar \u00f6ng\u00f6r\u00fclen zorunlu vital kapasitede ortalama 5,8% iyile\u015fme g\u00f6sterdi; bu art\u0131\u015f, Duchenne&#039;li yeti\u015fkinlerde y\u0131ll\u0131k yakla\u015f\u0131k 5%&#039;lik d\u00fc\u015f\u00fc\u015flerle do\u011fal seyirle de uyu\u015fmuyor. Di\u011fer t\u00fcm \u00f6l\u00e7\u00fcmler \u00e7al\u0131\u015fma s\u00fcresi boyunca sabit kald\u0131. Her iki \u00e7al\u0131\u015fmada da orta \u015fiddette veya daha y\u00fcksek \u015fiddette ila\u00e7la ili\u015fkili advers olaylar g\u00f6zlenmedi ve doz s\u0131n\u0131rlay\u0131c\u0131 toksisiteler meydana gelmedi.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"regardless-specific-dystrophin-mutation\">Belirli Ekzon Mutasyonundan Ba\u011f\u0131ms\u0131z<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">&quot;Satellos&#039;nin bilimsel kurucu ortaklar\u0131, v\u00fccudun do\u011fu\u015ftan gelen kas yenilenme s\u00fcrecinin Duchenne&#039;de ciddi \u015fekilde bozuldu\u011funu ilk fark edenlerdi. SAT-3247&#039;yi, v\u00fccudun kaslar\u0131 yeniden onar\u0131p in\u015fa edebilmesi i\u00e7in bu bozulmu\u015f yenilenme s\u00fcrecini yeniden ba\u015flatmay\u0131 hedefleyerek icat ettiler,&quot; dedi Satellos ba\u015fhekimi Dr. Wildon Farwell. &quot;Daha ileri hastal\u0131\u011f\u0131 olan yeti\u015fkinlerdeki bu erken etkililik belirtileri inan\u0131lmaz derecede cesaret verici ve klinik program\u0131m\u0131z\u0131 daha geni\u015f Duchenne toplulu\u011funa yaymam\u0131z\u0131 destekliyor. SAT-3247, bireyin spesifik distrofin mutasyonundan ba\u011f\u0131ms\u0131z olarak sonu\u00e7lar\u0131 iyile\u015ftirme potansiyeline sahip, kullan\u0131\u015fl\u0131 ve oral bir tedavi olarak tasarland\u0131.&quot;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"future-clinical-trial-plans\">Gelecekteki Klinik Ara\u015ft\u0131rma Planlar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Faz 1b \u00e7al\u0131\u015fmas\u0131na kat\u0131lan 20-27 ya\u015f aras\u0131 be\u015f yeti\u015fkin hasta, SAT-3247&#039;nin 11 ayl\u0131k a\u00e7\u0131k etiketli takip \u00e7al\u0131\u015fmas\u0131na kat\u0131lmaya davet edildi. Bu \u00e7al\u0131\u015fmaya 16-25 ya\u015f aras\u0131 DMD&#039;li ek erkekler de dahil edilecek. Bu \u00e7al\u0131\u015fman\u0131n birincil sonlan\u0131m noktalar\u0131, uzun vadeli g\u00fcvenlik ve tolerabilitenin yan\u0131 s\u0131ra SAT-3247&#039;nin biceps brachii kas\u0131ndaki ya\u011f oran\u0131 \u00fczerindeki etkisini de\u011ferlendirmektir. \u0130kincil sonlan\u0131m noktalar\u0131 aras\u0131nda SAT-3247&#039;nin ya\u011f oran\u0131 \u00fczerindeki etkisi ve kas kuvveti ve fonksiyonu \u00fczerindeki etkisi yer almaktad\u0131r. Kat\u0131l\u0131mc\u0131lar 3 ayl\u0131k takip ziyaretlerini tamamlad\u0131ktan sonra, \u015firket ilk ara sonu\u00e7lar\u0131 sunacakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Faz 1a\/b \u00e7al\u0131\u015fmas\u0131ndan elde edilen ilk g\u00fcvenlik ve etkinlik verilerine dayanarak, Satellos ayr\u0131ca, DMD&#039;li ayaktan \u00e7ocuklarda SAT-3247&#039;nin Faz 2 randomize, \u00e7ift k\u00f6r, plasebo kontroll\u00fc, k\u00fcresel, kavram kan\u0131tlama \u00e7al\u0131\u015fmas\u0131 planlamaktad\u0131r. Birincil sonlan\u0131m noktalar\u0131, SAT-3247&#039;nin g\u00fcvenli\u011fini ve tolerabilitesini ve kas kuvveti \u00fczerindeki etkisini de\u011ferlendirecektir. \u0130kincil sonlan\u0131m noktalar\u0131 ise SAT-3247&#039;nin kas kalitesi, i\u015flevi ve rejenerasyonu \u00fczerindeki etkisini de\u011ferlendirecektir. \u015eirket, bu \u00e7al\u0131\u015fmay\u0131 ilerletmek i\u00e7in yak\u0131n zamanda ABD ve k\u00fcresel \u00e7apta d\u00fczenleyici ba\u015fvurularda bulunmu\u015ftur.<\/p>","protected":false},"excerpt":{"rendered":"<p>Satellos Bioscience announced new data further demonstrating tolerability and initial efficacy of SAT-3247 in adults (aged 20-27 years) with Duchenne muscular dystrophy (Duchenne or DMD) at the 30th Annual Congress of the World Muscle Society in Vienna, Austria. Key Findings \u201cSatellos\u2019 new and updated clinical results from the 28-day clinical study in adults with Duchenne [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4777,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[254,251,252,253],"class_list":["post-4776","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-frank-gleeson","tag-sat-3247","tag-satellos","tag-satellos-bioscience"],"subtitle":"Satellos Bioscience'nin ara\u015ft\u0131rma a\u015famas\u0131ndaki SAT-3247 tedavisi, Duchenne hastalar\u0131na umut vermeye devam ediyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4776","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=4776"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4776\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4777"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4776"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4776"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4776"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}