{"id":13957,"date":"2019-10-21T13:39:45","date_gmt":"2019-10-21T17:39:45","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?page_id=13957"},"modified":"2022-11-10T05:44:01","modified_gmt":"2022-11-10T10:44:01","slug":"trikafta-fda-approval","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/trikafta-fda-approval\/","title":{"rendered":"Emily's Entourage'dan Kistik Fibrozis i\u00e7in \u00e7\u0131\u011f\u0131r a\u00e7an tedavinin FDA onay\u0131 ile ilgili a\u00e7\u0131klama"},"content":{"rendered":"<p><span style=\"font-weight: 400;\">Bug\u00fcn Kistik Fibrozis (KF) tedavileri ve t\u0131p tarihi a\u00e7\u0131s\u0131ndan muazzam bir g\u00fcn. FDA a\u00e7\u0131klad\u0131<\/span><a href=\"https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-new-breakthrough-therapy-cystic-fibrosis?fbclid=IwAR1jSmNevPR0f6XAk86GX4TWYNdIIKFsAyDsFSVGjVzH1NM8qWKgpxamkkE\"><span style=\"font-weight: 400;\"> merakla beklenen \u00fc\u00e7l\u00fc kombinasyon tedavisi Trikafta'y\u0131 onaylad\u0131, <\/span><\/a><span style=\"font-weight: 400;\">Vertex Pharmaceuticals taraf\u0131ndan geli\u015ftirilen Trifakta, en yayg\u0131n KF mutasyonu olan F508del'in en az bir kopyas\u0131na sahip olan 12 ya\u015f ve \u00fczeri KF pop\u00fclasyonunun 90%'si i\u00e7in geli\u015ftirilmi\u015ftir. Trifakta, iki nonsense mutasyonu veya di\u011fer tedavisi zor mutasyonlar\u0131 olanlara fayda sa\u011flamaz.<\/span><\/p>\n<p><span style=\"font-weight: 400;\">Trikafta iki nedenden \u00f6t\u00fcr\u00fc d\u00f6n\u00fcm noktas\u0131 niteli\u011finde bir geli\u015fmedir:\u00a0<\/span><\/p>\n<ul>\n<li style=\"font-weight: 400;\"><b>Etkinli\u011fi. <\/b><span style=\"font-weight: 400;\">Trikafta'n\u0131n faz 3 denemesi, ortalama akci\u011fer fonksiyonunu 13,8% art\u0131rarak t\u00fcm beklentileri a\u015fm\u0131\u015f ve akci\u011fer fonksiyonunu KF toplulu\u011fundaki bir\u00e7ok ki\u015finin on y\u0131llard\u0131r deneyimlemedi\u011fi bir seviyeye getirmi\u015ftir.\u00a0<\/span><\/li>\n<li style=\"font-weight: 400;\"><b>Kapsam\u0131.<\/b><span style=\"font-weight: 400;\"> Trikafta, en yayg\u0131n KF mutasyonuna (F508del) sahip KF toplulu\u011funun 90%'sine fayda sa\u011flamaktad\u0131r. Bug\u00fcne kadar, en etkili tedaviler KF pop\u00fclasyonunun yar\u0131s\u0131ndan daha az\u0131na fayda sa\u011flam\u0131\u015ft\u0131r ve hi\u00e7biri akci\u011fer fonksiyonunda bu oranda art\u0131\u015f g\u00f6stermemi\u015ftir.\u00a0<\/span><\/li>\n<\/ul>\n<p><span style=\"font-weight: 400;\">Trikafta'y\u0131 t\u0131bbi bir at\u0131l\u0131m olarak adland\u0131rmak b\u00fcy\u00fck bir yetersizlik olacakt\u0131r. Sekiz y\u0131l \u00f6nce, KF'nin alt\u0131nda yatan nedeni tedavi etmek i\u00e7in mutasyon hedefli hi\u00e7bir tedavi yoktu. Bug\u00fcn itibariyle, KF toplulu\u011funun 90%'sine fayda sa\u011flayan d\u00f6rt onayl\u0131 mutasyon hedefli tedavi bulunmaktad\u0131r. Bu ola\u011fan\u00fcst\u00fc bir geli\u015fmedir, ancak ayn\u0131 zamanda <\/span><span style=\"font-weight: 400;\">olarak <strong>Bu da KF toplumunun 10%'lik bir k\u0131sm\u0131n\u0131n -ki nonsense mutasyonuna sahip olanlar en b\u00fcy\u00fck grubu olu\u015fturmaktad\u0131r- herhangi bir tedaviden yoksun kald\u0131\u011f\u0131n\u0131 hat\u0131rlatmaktad\u0131r.<\/strong>\u00a0<\/span><\/p>\n<p><span style=\"font-weight: 400;\">Emily's Entourage'\u0131 2011 y\u0131l\u0131nda kurduk \u00e7\u00fcnk\u00fc ufukta Trikafta gibi geli\u015fmeler g\u00f6rd\u00fck ve Emily gibi KF'nin sa\u00e7ma mutasyonlar\u0131na sahip olanlar\u0131n bu ilerlemelerin hi\u00e7birinden yararlanamayaca\u011f\u0131n\u0131 fark ettik. KF'nin sa\u00e7ma mutasyonlar\u0131na sahip insanlar, hastal\u0131klar\u0131 devam ederken geride b\u0131rak\u0131l\u0131yorlard\u0131. Bu kaderi kabul etmeyi reddettik.\u00a0<\/span><\/p>\n<p><span style=\"font-weight: 400;\">Bug\u00fcn, KF toplulu\u011fu i\u00e7in b\u00fcy\u00fck bir zaferi kutluyoruz. Trikafta'n\u0131n KF pop\u00fclasyonunun 90%'si i\u00e7in sa\u011flad\u0131\u011f\u0131 umuttan ve bunun di\u011fer pek \u00e7ok nadir hastal\u0131k i\u00e7in a\u00e7t\u0131\u011f\u0131 yoldan dolay\u0131 \u00e7ok mutluyuz. Ayr\u0131ca 90%'nin yeterli olmad\u0131\u011f\u0131na her zamankinden daha g\u00fc\u00e7l\u00fc bir \u015fekilde inan\u0131yoruz. Nonsense mutasyonlar\u0131 olanlar da dahil olmak \u00fczere KF toplulu\u011funun 100%'sine ilerlemeyi geni\u015fletmek ve ila\u00e7 geli\u015ftirme s\u00fcrecinde yenilikler yaparak bunu daha \u00f6nce hi\u00e7 yap\u0131lmad\u0131\u011f\u0131 kadar h\u0131zl\u0131 yapmam\u0131z\u0131 sa\u011flamak i\u00e7in kararl\u0131l\u0131\u011f\u0131m\u0131z\u0131 ve lazer oda\u011f\u0131m\u0131z\u0131 s\u00fcrd\u00fcr\u00fcyoruz.\u00a0<\/span><\/p>\n<p><span style=\"font-weight: 400;\">Trikafta devrim niteli\u011finde olsa da <\/span><span style=\"font-weight: 400;\">de\u011fil <\/span><span style=\"font-weight: 400;\">bir tedavi. Hala mutasyon hedefli tedavileri olmayan 10% ve Trikafta'dan yararlanan 90% i\u00e7in KF'nin ortadan kald\u0131r\u0131ld\u0131\u011f\u0131 veya \"iyile\u015ftirildi\u011fi\" alg\u0131s\u0131na kar\u015f\u0131 uyan\u0131k kalmal\u0131y\u0131z, \u00e7\u00fcnk\u00fc bu yeni tedavi KF i\u00e7in bir tedavi de\u011fil \"kontrol\" i\u015flevi g\u00f6rmektedir.\u00a0<\/span><\/p>\n<p><span style=\"font-weight: 400;\">Emily's Entourage'\u0131n \u00e7al\u0131\u015fmalar\u0131, at\u0131l\u0131mlar yap\u0131lana ve KF'li herkes i\u00e7in bir tedavi bulunana kadar t\u00fcm h\u0131z\u0131yla devam ediyor.\u00a0<\/span><\/p>\n<p><b>Emily'nin \u00c7evresi Hakk\u0131nda<br \/>\n<\/b><span style=\"font-weight: 400;\">Emily's Entourage (EE), \u00f6l\u00fcmc\u00fcl bir genetik akci\u011fer hastal\u0131\u011f\u0131 olan Kistik Fibrozis'in nadir g\u00f6r\u00fclen nonsense mutasyonlar\u0131na y\u00f6nelik \u00e7\u0131\u011f\u0131r a\u00e7an tedavileri ve tedaviyi h\u0131zland\u0131rmak i\u00e7in 2011 y\u0131l\u0131ndan bu yana milyonlarca dolar toplayan yenilik\u00e7i bir 501(c)3 vakf\u0131d\u0131r. Kurulu\u015f, ileri evre Kistik Fibrozis hastas\u0131 gen\u00e7 bir kad\u0131n olan Emily Kramer-Golinkoff ve onun sad\u0131k arkada\u015flar\u0131 ve ailesi taraf\u0131ndan kurulmu\u015ftur.\u00a0<\/span><\/p>\n<p><span style=\"font-weight: 400;\">Emily'nin durumunun aciliyeti, sadece Kistik Fibrozis (KF) hastalar\u0131na de\u011fil, ayn\u0131 zamanda kas distrofisi ve kanser t\u00fcrleri de dahil olmak \u00fczere sa\u00e7ma mutasyonlar\u0131n neden oldu\u011fu \u00e7ok \u00e7e\u015fitli genetik hastal\u0131klardan muzdarip insanlara yard\u0131mc\u0131 olabilecek devrim niteli\u011findeki ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirmeyi te\u015fvik ediyor. Sa\u00e7ma mutasyonlar, KF'liler aras\u0131nda nadir g\u00f6r\u00fclmekle birlikte, t\u00fcm genetik hastal\u0131klar\u0131n yakla\u015f\u0131k 12%'sinin nedenidir ve d\u00fcnya \u00e7ap\u0131nda 30 milyon insan\u0131 etkilemektedir.<\/span><\/p>\n<p><i><span style=\"font-weight: 400;\">Medya ile ilgili sorular\u0131n\u0131z i\u00e7in l\u00fctfen K\u0131demli Pazarlama ve Program Koordinat\u00f6r\u00fc Kira Rentas'a \u015fu adresten ula\u015f\u0131n <\/span><\/i><a href=\"mailto:kira@emilysentourage.org\" target=\"_blank\" rel=\"noopener noreferrer\"><i><span style=\"font-weight: 400;\">kira@emilysentourage.org <\/span><\/i><\/a><i><span style=\"font-weight: 400;\">veya 631-902-1993. <\/span><\/i><\/p>","protected":false},"excerpt":{"rendered":"<p>Bug\u00fcn Kistik Fibrozis (KF) tedavileri ve t\u0131p tarihi a\u00e7\u0131s\u0131ndan \u00e7ok \u00f6nemli bir g\u00fcn. FDA, Vertex Pharmaceuticals taraf\u0131ndan geli\u015ftirilen ve merakla beklenen \u00fc\u00e7l\u00fc kombinasyon tedavisi Trikafta'n\u0131n, en yayg\u0131n KF vir\u00fcs\u00fcn\u00fcn en az bir kopyas\u0131na sahip olan 12 ya\u015f ve \u00fczeri KF pop\u00fclasyonunun 90%'si i\u00e7in onayland\u0131\u011f\u0131n\u0131 duyurdu... <\/p>\n<div class=\"clear\"><\/div>\n<p><a href=\"https:\/\/www.emilysentourage.org\/tr\/trikafta-fda-approval\/\" class=\"excerpt-read-more\">Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1704,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"","_seopress_titles_desc":"","_seopress_robots_index":"","_seopress_robots_follow":"","_seopress_robots_imageindex":"","_seopress_robots_snippet":"","_seopress_robots_primary_cat":"none","_seopress_robots_breadcrumbs":"","_seopress_robots_freeze_modified_date":"","_seopress_robots_custom_modified_date":"","_seopress_robots_canonical":"","_seopress_social_fb_title":"","_seopress_social_fb_desc":"","_seopress_social_fb_img":"","_seopress_social_fb_img_attachment_id":0,"_seopress_social_fb_img_width":0,"_seopress_social_fb_img_height":0,"_seopress_social_twitter_title":"","_seopress_social_twitter_desc":"","_seopress_social_twitter_img":"","_seopress_social_twitter_img_attachment_id":0,"_seopress_social_twitter_img_width":0,"_seopress_social_twitter_img_height":0,"_seopress_redirections_value":"","_seopress_redirections_enabled":"","_seopress_redirections_enabled_regex":"","_seopress_redirections_logged_status":"both","_seopress_redirections_param":"","_seopress_redirections_type":301,"_seopress_analysis_target_kw":"","_seopress_news_disabled":"","_seopress_video_disabled":"","_seopress_video":[{"url":"","title":"","desc":"","thumbnail":"","duration":"","rating":"","view_count":"","tag":"","cat":""}],"_seopress_pro_schemas_manual":[{"_seopress_pro_rich_snippets_type":"none"}],"_seopress_pro_rich_snippets_disable_all":"","_seopress_pro_rich_snippets_disable":[],"_seopress_pro_schemas":[],"_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"_jetpack_newsletter_access":"","_jetpack_dont_email_post_to_subs":false,"_jetpack_newsletter_tier_id":0,"_jetpack_memberships_contains_paywalled_content":false,"_jetpack_feature_clip_id":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_post_was_ever_published":false},"categories":[178],"tags":[],"class_list":["post-13957","post","type-post","status-publish","format-standard","hentry","category-press-releases"],"acf":[],"jetpack_featured_media_url":"","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/13957","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/users\/1704"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=13957"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/13957\/revisions"}],"predecessor-version":[{"id":17618,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/13957\/revisions\/17618"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=13957"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=13957"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=13957"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}