{"id":8848,"date":"2026-10-05T19:00:36","date_gmt":"2026-10-05T16:00:36","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8848"},"modified":"2026-10-05T19:09:42","modified_gmt":"2026-10-05T16:09:42","slug":"deramiocel-hope-3-24-month-results-duchenne","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/deramiocel-hope-3-24-month-results-duchenne\/","title":{"rendered":"Deramiocel, Duchenne\u2019de 24 Ayl\u0131k HOPE-3 \u00c7al\u0131\u015fmas\u0131nda Olumlu Sonu\u00e7lar G\u00f6sterdi"},"content":{"rendered":"

Capricor Therapeutics, Duchenne kas distrofisi (DMD) hastalar\u0131nda Deramiocel'i de\u011ferlendiren HOPE-3 \u00e7al\u0131\u015fmas\u0131ndan yeni sonu\u00e7lar bildirdi.<\/strong> \u00dcst ekstremite fonksiyonuna odaklanan 24 ayl\u0131k a\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131 verileri, plasebo ile bir y\u0131l tedavi g\u00f6rd\u00fckten sonra Deramiocel tedavisine ba\u015flayan hastalar\u0131n, tedavinin ilk y\u0131l\u0131nda \u00fcst ekstremite fonksiyonlar\u0131nda \u00f6nemli \u00f6l\u00e7\u00fcde daha yava\u015f bir gerileme ya\u015fad\u0131\u011f\u0131n\u0131 g\u00f6stermi\u015ftir.<\/p>\n\n\n\n

Yeni bulgular 2026 D\u00fcnya Kas Toplulu\u011fu Kongresi'nde sunuldu. HOPE-3 \u00e7al\u0131\u015fmas\u0131, Duchenne kas distrofisi olan hastalarda Deramiocel'i de\u011ferlendiren bir Faz 3 klinik \u00e7al\u0131\u015fmas\u0131d\u0131r. \u00c7al\u0131\u015fman\u0131n randomize, plasebo kontroll\u00fc b\u00f6l\u00fcm\u00fcne ba\u015flang\u0131\u00e7ta toplam 106 hasta dahil edildi. Bu d\u00f6nemi tamamlayan hastalar, a\u00e7\u0131k etiketli bir uzatma d\u00f6neminde Deramiocel almaya devam edebilirler. Daha Fazla Bilgi Edinin: Deramiocel Nedir?<\/a><\/p>\n\n\n\n

Deramiocel'e Ba\u015fland\u0131ktan Sonra \u00dcst Ekstremitedeki Gerileme Yava\u015flad\u0131<\/h2>\n\n\n\n

En \u00f6nemli bulgulardan biri, ilk y\u0131l plasebo alan ve daha sonra Deramiocel'ye ge\u00e7en hastalardan elde edildi.<\/p>\n\n\n\n

Bu hastalar, plasebo tedavisine ba\u015flad\u0131klar\u0131 ilk y\u0131l boyunca PUL 2.0 toplam puanlar\u0131nda ortalama 2,05 puanl\u0131k bir d\u00fc\u015f\u00fc\u015f ya\u015fad\u0131lar.<\/strong> Deramiocel'in ba\u015flamas\u0131n\u0131n ard\u0131ndan gelen y\u0131l boyunca, ortalama d\u00fc\u015f\u00fc\u015fleri 0,49 puana geriledi.<\/p>\n\n\n\n

Capricor'a g\u00f6re bu, hastalar\u0131n plasebo ald\u0131klar\u0131 \u00f6nceki y\u0131la k\u0131yasla \u00fcst ekstremite kay\u0131p oran\u0131nda 76%'lik bir azalmay\u0131 temsil etmektedir.<\/strong><\/p>\n\n\n\n

\u00dcst ekstremite fonksiyonu, Duchenne kas distrofisi olan ki\u015filer i\u00e7in \u00f6zellikle \u00f6nemlidir \u00e7\u00fcnk\u00fc ba\u011f\u0131ms\u0131zl\u0131k ve g\u00fcnl\u00fck aktivitelerle yak\u0131ndan ba\u011flant\u0131l\u0131d\u0131r.<\/p>\n\n\n\n

Ba\u015flang\u0131\u00e7tan \u0130tibaren Deramiocel Alan Hastalar<\/h2>\n\n\n\n

\u00c7al\u0131\u015fmada ayr\u0131ca, denemenin ba\u015flang\u0131c\u0131ndan itibaren Deramiocel alan hastalar da takip edildi.<\/p>\n\n\n\n

Bu grupta, ortalama PUL 2.0 puan\u0131 ilk y\u0131lda 0,95 puan, ikinci y\u0131lda ise 0,89 puan azald\u0131.<\/strong> \u0130ki y\u0131l boyunca elde edilen benzer sonu\u00e7lar, yava\u015flayan d\u00fc\u015f\u00fc\u015f\u00fcn tedavinin ikinci y\u0131l\u0131nda da devam etti\u011fini g\u00f6stermektedir.<\/p>\n\n\n\n

Do\u011fal Hastal\u0131k Seyriyle Kar\u015f\u0131la\u015ft\u0131rma<\/h2>\n\n\n\n

24. ayda, her iki grupta da \u00fcst ekstremite fonksiyonundaki gerileme, do\u011fal seyir modellerinin \u00f6ng\u00f6rd\u00fc\u011f\u00fcnden daha azd\u0131.<\/p>\n\n\n\n

\u0130lk y\u0131l plasebo, ikinci y\u0131l ise Deramiocel alan hastalarda g\u00f6zlemlenen de\u011fi\u015fim -3,76 puan olurken, do\u011fal seyrin \u00f6ng\u00f6rd\u00fc\u011f\u00fc d\u00fc\u015f\u00fc\u015f -5,35 puand\u0131.<\/strong><\/p>\n\n\n\n

Her iki y\u0131l boyunca Deramiocel alan hastalarda g\u00f6zlemlenen de\u011fi\u015fim -3,42 puan olurken, tahmin edilen d\u00fc\u015f\u00fc\u015f -5,88 puand\u0131.<\/strong><\/p>\n\n\n\n

Ancak Capricor, bu do\u011fal seyir kar\u015f\u0131la\u015ft\u0131rmalar\u0131n\u0131n yaln\u0131zca tan\u0131mlay\u0131c\u0131 nitelikte oldu\u011funu ve hasta e\u015fle\u015ftirmesine veya istatistiksel testlere dayanmad\u0131\u011f\u0131n\u0131 belirtti.<\/p>\n\n\n\n

Bu Sonu\u00e7lar\u0131n Anlam\u0131<\/h2>\n\n\n\n

24 ayl\u0131k sonu\u00e7lar, Deramiocel'in Duchenne kas distrofisi olan ki\u015filerde \u00fcst ekstremite fonksiyon kayb\u0131n\u0131 yava\u015flatabilece\u011fine dair ek kan\u0131tlar sunmaktad\u0131r. En dikkat \u00e7ekici bulgu, plasebodan Deramiocel'ye ge\u00e7en hastalarda gerileme oran\u0131ndaki azalmad\u0131r.<\/p>\n\n\n\n

Ayn\u0131 zamanda, a\u00e7\u0131k etiketli uzatma \u00e7al\u0131\u015fmas\u0131, 24. ayda resmi istatistiksel kar\u015f\u0131la\u015ft\u0131rmalar i\u00e7in tasarlanmam\u0131\u015f veya yeterli g\u00fcce sahip de\u011fildi. Bu nedenle, sonu\u00e7lar \u00e7al\u0131\u015fma tasar\u0131m\u0131n\u0131n s\u0131n\u0131rlamalar\u0131 dahilinde yorumlanmal\u0131d\u0131r.<\/p>\n\n\n\n

Capricor, bu verileri Deramiocel Biyolojik \u00dcr\u00fcn Lisans Ba\u015fvurusuna (BLA) yap\u0131lan \u00f6nemli bir de\u011fi\u015fikli\u011fin par\u00e7as\u0131 olarak sunmu\u015ftur. FDA numaral\u0131 ba\u015fvurunun mevcut PDUFA hedef i\u015flem tarihi 22 Kas\u0131m 2026'd\u0131r.<\/strong><\/p>\n\n\n\n

Daha Fazlas\u0131n\u0131 Ke\u015ffedin<\/strong>:\u00a0Duchenne Klinik Ara\u015ft\u0131rma Yerleri Haritas\u0131<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Capricor Therapeutics has reported new results from the HOPE-3 study evaluating Deramiocel in people with Duchenne muscular dystrophy (DMD). The 24-month open-label extension data focused on Upper Limb Function and showed that patients who started Deramiocel after one year on placebo experienced a significantly slower rate of upper limb decline during their first year of […]<\/p>\n","protected":false},"author":3,"featured_media":8849,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[90,86,87,85,88,926,923],"class_list":["post-8848","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-cap-1002","tag-capricor","tag-capricor-therapeutics","tag-deramiocel","tag-fda","tag-hope-3","tag-wms-2026"],"subtitle":"Capricor Therapeutics, Duchenne kas distrofisi hastalar\u0131nda Deramiocel i\u00e7in 24 ayl\u0131k HOPE-3 \u00e7al\u0131\u015fmas\u0131nda cesaret verici sonu\u00e7lar bildirdi. Veriler, hastalar\u0131n plasebodan tedaviye ge\u00e7mesinden sonra \u00fcst ekstremite fonksiyonunda daha yava\u015f bir d\u00fc\u015f\u00fc\u015f oldu\u011funu ve 76% oran\u0131nda bir azalma oldu\u011funu g\u00f6stermektedir.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8848","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\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=8848"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8848\/revisions"}],"predecessor-version":[{"id":8851,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8848\/revisions\/8851"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8849"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8848"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8848"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8848"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}