{"id":3240,"date":"2025-03-17T16:51:32","date_gmt":"2025-03-17T13:51:32","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3240"},"modified":"2025-03-17T16:59:56","modified_gmt":"2025-03-17T13:59:56","slug":"capricor-therapeutics-announces-positive-data-demonstrating-long-term-efficacy-of-deramiocel-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/capricor-therapeutics-announces-positive-data-demonstrating-long-term-efficacy-of-deramiocel-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Capricor Therapeutics, Duchenne Musk\u00fcler Distrofi Tedavisinde Deramiocel&#039;nin Uzun Vadeli Etkinli\u011fini G\u00f6steren Olumlu Verileri Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Nadir hastal\u0131klar\u0131n tedavisi i\u00e7in d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc h\u00fccre ve ekzosom bazl\u0131 tedaviler geli\u015ftiren bir biyoteknoloji \u015firketi olan Capricor Therapeutics, bug\u00fcn \u015firketin devam eden HOPE-2 a\u00e7\u0131k etiketli uzatma (&quot;OLE&quot;) klinik denemesinden elde edilen olumlu uzun vadeli sonu\u00e7lar\u0131 duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu sonu\u00e7lar, \u015firketin \u00f6nc\u00fc varl\u0131\u011f\u0131 olan deramiocel&#039;in, Duchenne kas distrofisi (&quot;DMD&quot;) hastalar\u0131nda hastal\u0131\u011f\u0131n ilerlemesini yava\u015flatma ve \u00fcst ekstremite fonksiyonunu koruma potansiyeline sahip oldu\u011funu g\u00f6stermektedir. 16-19 Mart tarihleri aras\u0131nda Dallas, Teksas&#039;ta ger\u00e7ekle\u015fen bu y\u0131lki Kas Distrofisi Derne\u011fi Klinik ve Bilimsel Konferans\u0131, verileri son dakika posteri olarak sundu.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Capricor Therapeutics Deramiocel Klinik Denemeler<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Kohort e\u015fle\u015ftirilmi\u015f harici kar\u015f\u0131la\u015ft\u0131rma analizinde, \u00e7al\u0131\u015fma \u00fc\u00e7 y\u0131l boyunca deramiocel ile tedavi edilen hastalar\u0131n \u00dcst Uzuv Performans\u0131 (PUL 2.0) toplam puan\u0131nda ortalama 3,46 puanl\u0131k bir d\u00fc\u015f\u00fc\u015f ya\u015fad\u0131\u011f\u0131n\u0131, harici kar\u015f\u0131la\u015ft\u0131rma grubunda ise 7,19 puanl\u0131k bir d\u00fc\u015f\u00fc\u015f oldu\u011funu g\u00f6sterdi (p=0,019). Bu, hastal\u0131\u011f\u0131n ilerlemesinin % 52 yava\u015flamas\u0131na e\u015fde\u011ferdir ve deramiocel&#039;in potansiyel uzun vadeli terap\u00f6tik dayan\u0131kl\u0131l\u0131\u011f\u0131n\u0131 g\u00fc\u00e7lendirir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ek bulgular \u015funlar\u0131 i\u00e7erir:<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Tedavi etkisi y\u0131ldan y\u0131la artmaktad\u0131r<\/strong> \u2013 Deramiocel kullanan hastalarda hastal\u0131\u011f\u0131n ilerlemesinde azalma g\u00f6r\u00fcld\u00fc; 1. y\u0131lda ortalama y\u0131ll\u0131k PUL 2.0 d\u00fc\u015f\u00fc\u015f\u00fc 1.8 puan, 2. y\u0131lda 1.2 puan ve 3. y\u0131lda 1.1 puan oldu.<\/li>\n\n\n\n<li><strong>Potansiyel hastal\u0131\u011f\u0131 de\u011fi\u015ftirici etkiler<\/strong> \u2013 1 y\u0131ll\u0131k tedavi bo\u015flu\u011fu s\u0131ras\u0131nda, ba\u015flang\u0131\u00e7ta deramiocel&#039;e randomize edilen hastalarda, tedavi edilmeyen hastalara (y\u0131lda 3,7 puan) k\u0131yasla daha yava\u015f bir d\u00fc\u015f\u00fc\u015f oran\u0131 (y\u0131lda 2,8 puan) g\u00f6r\u00fcld\u00fc.<\/li>\n\n\n\n<li><strong>Uygun g\u00fcvenlik profili<\/strong> \u2013 Deramiocel iyi tolere edildi, yeni g\u00fcvenlik sinyalleri tespit edilmedi ve uzun vadede olumlu bir fayda-risk profili s\u00fcrd\u00fcrmeye devam ediyor.<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Positive long-term results from the company&#8217;s ongoing HOPE-2 open label extension (&#8220;OLE&#8221;) clinical trial were announced today by Capricor Therapeutics, a biotechnology company that develops transformative cell and exosome-based therapeutics for the treatment of rare diseases. These results show that the company&#8217;s lead asset, deramiocel, has the potential to slow the progression of the disease [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3241,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[90,87,89,85,301,297],"class_list":["post-3240","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-cap-1002","tag-capricor-therapeutics","tag-cardiomyopathy","tag-deramiocel","tag-hope-2","tag-muscular-dystrophy-association"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3240","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=3240"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3240\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3241"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3240"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3240"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3240"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}