{"id":23287,"date":"2024-09-30T09:32:11","date_gmt":"2024-09-30T13:32:11","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=23287"},"modified":"2024-09-30T12:07:22","modified_gmt":"2024-09-30T16:07:22","slug":"paul-mccray-jr-md-announced-as-recipient-of-emilys-entourage-2024-trailblazer-award","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/paul-mccray-jr-md-announced-as-recipient-of-emilys-entourage-2024-trailblazer-award\/","title":{"rendered":"Paul McCray, Jr, MD, Emily's Entourage 2024 Trailblazer \u00d6d\u00fcl\u00fcn\u00fcn Sahibi Olarak A\u00e7\u0131kland\u0131"},"content":{"rendered":"
\"\"

Iowa \u00dcniversitesi'nde pediatri-pulmoner t\u0131p profes\u00f6r\u00fc olan Paul McCray, Jr, Boston, Massachusetts'te d\u00fczenlenen 2024 Kuzey Amerika Kistik Fibrozis Konferans\u0131 (NACFC) s\u0131ras\u0131nda 2024 Emily's Entourage Trailblazer \u00d6d\u00fcl\u00fc'ne lay\u0131k g\u00f6r\u00fcld\u00fc.<\/p><\/div>\n

\u00d6d\u00fcl, Dr. McCray'i gen tedavilerindeki \u00f6nc\u00fc \u00e7al\u0131\u015fmalar\u0131 ve mevcut CFTR mod\u00fclat\u00f6rlerinden yararlanamayan kistik fibrozis toplulu\u011funun son 10%'sine adanm\u0131\u015fl\u0131\u011f\u0131 nedeniyle takdir etmektedir.<\/i><\/p>\n

Lower Merion, PA - 30 Eyl\u00fcl 2024<\/b> - Kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve tedavi ara\u015ft\u0131rmalar\u0131n\u0131 h\u0131zland\u0131ran bir 501(c)(3) vakf\u0131 olan Emily's Entourage (EE), Iowa \u00dcniversitesi'nde pediatri-pulmoner t\u0131p profes\u00f6r\u00fc olan Paul McCray, Jr'\u0131 prestijli 2024 Trailblazer \u00d6d\u00fcl\u00fc'ne lay\u0131k g\u00f6rd\u00fc. Bu \u00f6d\u00fcl, KF toplulu\u011fu i\u00e7in hayat kurtaran ara\u015ft\u0131rmalar\u0131 ilerletmede yenilik, cesaret ve azim g\u00f6steren liderleri kutlamaktad\u0131r.<\/p>\n

Dr. McCray, EE'nin sadece davetlilerin kat\u0131ld\u0131\u011f\u0131 resepsiyonunda onurland\u0131r\u0131ld\u0131. Kuzey Amerika Kistik Fibrozis Konferans\u0131 (NACFC)<\/a> Cuma g\u00fcn\u00fc, 27 Eyl\u00fcl 2024, Boston, Massachusetts'te. Resepsiyon, d\u00fcnyan\u0131n \u00f6nde gelen yakla\u015f\u0131k 200 KF ara\u015ft\u0131rmac\u0131s\u0131n\u0131, bak\u0131m sa\u011flay\u0131c\u0131s\u0131n\u0131 ve sekt\u00f6r liderini bir araya getirdi.<\/p>\n

Emily's Entourage'\u0131n kurucu orta\u011f\u0131 Emily Kramer-Golinkoff, MBE, \"Paul, KF bak\u0131m\u0131n\u0131n gelece\u011fini d\u00f6n\u00fc\u015ft\u00fcren vizyoner, \u015fefkatli bir ara\u015ft\u0131rmac\u0131 ve hekimdir\" dedi. \"Gen terapisindeki son teknoloji \u00e7al\u0131\u015fmalar\u0131 ve EE ile ortakl\u0131\u011f\u0131 sayesinde, mevcut CFTR mod\u00fclat\u00f6r tedavilerinden fayda g\u00f6rmeyen son 10%'deki insanlar i\u00e7in \u00e7\u0131\u011f\u0131r a\u00e7an tedavilere kap\u0131 a\u00e7\u0131yor. Kendisini bu y\u0131lki Trailblazer \u00d6d\u00fcl\u00fc ile onurland\u0131rmaktan heyecan duyuyoruz.\"<\/p>\n

Dr. McCray'in ara\u015ft\u0131rmalar\u0131, KF dahil olmak \u00fczere akci\u011fer hastal\u0131klar\u0131 i\u00e7in genetik tedavilerin geli\u015ftirilmesinde \u00e7ok \u00f6nemli bir rol oynam\u0131\u015ft\u0131r. \u00c7al\u0131\u015fmalar\u0131, solunum epitel h\u00fccrelerine gen iletim sistemlerini geli\u015ftirmeye odaklanmaktad\u0131r. May\u0131s 2024'te Dr. McCray'in ekibi EE'nin $858,932 tutar\u0131ndaki ilk i\u015fbirli\u011fi hibesi Kistik Fibrozis Tedavilerinde Hedefe Y\u00f6nelik Hava Yolu \u0130letimine Yeni Yakla\u015f\u0131mlar<\/i><\/a>.<\/i> Proje, genetik tedavilerin insan hava yolu epitellerine ula\u015ft\u0131r\u0131lmas\u0131 i\u00e7in en etkili AAV kapsid varyantlar\u0131n\u0131 belirlemeyi ve bunlar\u0131n etkinli\u011fini de\u011ferlendirmeyi ama\u00e7lamaktad\u0131r. Bu geli\u015fmeler, uygun olmayan mutasyonlar\u0131, advers reaksiyonlar\u0131 veya mevcut tedavilere suboptimal yan\u0131tlar\u0131 olan KF hastalar\u0131 i\u00e7in kritik \u00f6neme sahiptir.<\/p>\n

Dr. McCray, laboratuvar tezgah\u0131ndaki \u00e7al\u0131\u015fmalar\u0131na ek olarak, \u015fu \u015firketin bilimsel kurucusu ve dan\u0131\u015fman\u0131d\u0131r Spirovant Bilimleri<\/a> - eski ad\u0131yla Talee Bio - EE taraf\u0131ndan tasarlanan ve tohum finansman\u0131 sa\u011flanan, kistik fibroz i\u00e7in yeni gen terapisi yakla\u015f\u0131mlar\u0131 geli\u015ftirmeye odaklanan bir biyofarmas\u00f6tik \u015firket.<\/p>\n

EE'nin Ba\u015f Bilimsel Sorumlusu PhD Chandra Ghose, \"Dr. McCray'in gen terapisindeki \u00f6nc\u00fc \u00e7al\u0131\u015fmalar\u0131, ya\u015famlar\u0131 d\u00f6n\u00fc\u015ft\u00fcrmek i\u00e7in ola\u011fan\u00fcst\u00fc bir umut vaat ediyor\" dedi. \"\u00d6zellikle, hedeflenen hava yolu da\u011f\u0131t\u0131m\u0131na odaklanmas\u0131, mevcut tedavilerin ula\u015famad\u0131\u011f\u0131 mutasyonlara sahip olanlar i\u00e7in yeni bir umut sunuyor ve bizi KF toplulu\u011funun acilen ihtiya\u00e7 duydu\u011fu d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc ilerlemelere yakla\u015ft\u0131r\u0131yor.\"<\/p>\n

\"\"

Kevin Foskett, PhD, Emily's Entourage Bilimsel Dan\u0131\u015fma Kurulu Ba\u015fkan\u0131; Paul McCray, Jr, MD, 2024 Emily's Entourage Trailblazer \u00d6d\u00fcl\u00fc sahibi; ve Chandra Ghose, PhD, Emily's Entourage Bilimsel Ba\u015f Sorumlusu Boston'daki NACFC'de d\u00fczenlenen 7. Y\u0131ll\u0131k Emily's Entourage Resepsiyonunda.<\/p><\/div>\n

KF ara\u015ft\u0131rmalar\u0131nda uluslararas\u0131 d\u00fczeyde tan\u0131nan bir lider olan Dr. McCray'in \u00e7al\u0131\u015fmalar\u0131, genetik akci\u011fer hastal\u0131klar\u0131 i\u00e7in gen terapisinin \u00f6n saflar\u0131nda yer almaktad\u0131r. Laboratuvar\u0131, hava yolu epitel h\u00fccrelerinde gen eklenmesi ve d\u00fczenlenmesi i\u00e7in viral ve viral olmayan vekt\u00f6rler geli\u015ftirmekte ve \u00e7ocukluk \u00e7a\u011f\u0131nda ba\u015flayan KF ve primer siliyer diskinezi gibi durumlar\u0131 ele almaktad\u0131r.<\/p>\n

Ara\u015ft\u0131rmalar\u0131n\u0131n \u00f6tesinde Dr. McCray, KF'li insanlar ve aileleri i\u00e7in kendini adam\u0131\u015f bir ak\u0131l hocas\u0131 ve savunucudur. Son 10%'ye olan sars\u0131lmaz ba\u011fl\u0131l\u0131\u011f\u0131, hayat de\u011fi\u015ftiren tedavileri ger\u00e7e\u011fe d\u00f6n\u00fc\u015ft\u00fcrmek i\u00e7in geleneksel s\u0131n\u0131rlar\u0131n \u00f6tesine ge\u00e7enleri onurland\u0131ran Trailblazer \u00d6d\u00fcl\u00fc'n\u00fcn ruhunu \u00f6rneklemektedir.<\/p>\n

\"Emily's Entourage'dan 2024 Trailblazer \u00d6d\u00fcl\u00fc'n\u00fc almaktan b\u00fcy\u00fck onur duyuyorum. Kistik fibrozis i\u00e7in genetik tedavileri ilerletmeye \u00e7al\u0131\u015f\u0131rken bu takdir benim ve ekibim i\u00e7in \u00e7ok \u015fey ifade ediyor,\" dedi Dr. McCray. \"Emily's Entourage yenilik\u00e7i ara\u015ft\u0131rmalar\u0131n y\u00fcr\u00fct\u00fclmesinde paha bi\u00e7ilmez bir ortak oldu ve birlikte, hala at\u0131l\u0131m bekleyenlere hayatlar\u0131n\u0131 de\u011fi\u015ftirecek tedaviler sunmay\u0131 hedefliyoruz. KF toplulu\u011funun son 10%'si i\u00e7in yaratt\u0131\u011f\u0131m\u0131z umuttan ger\u00e7ekten ilham al\u0131yorum.\"<\/p>\n

 <\/p>\n

Emily'nin \u00c7evresi Hakk\u0131nda<\/b><\/p>\n

Emily's Entourage, mevcut mutasyon hedefli tedavilerden yararlanamayan kistik fibrozis (KF) pop\u00fclasyonunun son 10%'sindeki bireyler i\u00e7in yeni tedaviler ve bir tedavi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3't\u00fcr. Emily's Entourage 2011 y\u0131l\u0131ndan bu yana milyonlarca dolarl\u0131k ara\u015ft\u0131rma hibesi vermi\u015f, \u015fu anda sat\u0131n al\u0131nan bir KF gen terapisi \u015firketini kurmu\u015f, klinik deneylere kat\u0131l\u0131m\u0131 h\u0131zland\u0131rmak i\u00e7in bir hasta veri taban\u0131 ve klinik deney e\u015fle\u015ftirme program\u0131 geli\u015ftirmi\u015f ve d\u00fcnya \u00e7ap\u0131nda y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirme \u00e7abalar\u0131na \u00f6nc\u00fcl\u00fck etmi\u015ftir. Kurulu\u015f, New York Times, STAT, CNN, People ve daha fazlas\u0131 dahil olmak \u00fczere ulusal medyada yer alm\u0131\u015ft\u0131r. Daha fazla bilgi i\u00e7in emilysentourage.org<\/a>.<\/p>\n

\u00a0<\/i><\/p>\n

Medya \u0130leti\u015fim<\/b><\/p>\n

Hannah Kleckner<\/p>\n

Pazarlama ve \u0130leti\u015fim Direkt\u00f6r\u00fc<\/p>\n

hannah@emilysentourage.org<\/p>","protected":false},"excerpt":{"rendered":"

\u00d6d\u00fcl, Dr. McCray'i gen tedavilerindeki \u00f6nc\u00fc \u00e7al\u0131\u015fmalar\u0131 ve mevcut CFTR mod\u00fclat\u00f6rlerinden yararlanamayan kistik fibrozis toplulu\u011funun son 10%'sine olan ba\u011fl\u0131l\u0131\u011f\u0131 nedeniyle takdir etmektedir. Lower Merion, PA - 30 Eyl\u00fcl 2024 - Emily's Entourage (EE), yeni tedaviler ve kistik fibrozis hastal\u0131\u011f\u0131n\u0131n tedavisi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran bir 501(c)(3) vakf\u0131... <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1719,"featured_media":23296,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_titles_title":"%%post_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":"","_monsterinsights_skip_tracking":false,"_jetpack_newsletter_access":"","_jetpack_dont_email_post_to_subs":false,"_jetpack_newsletter_tier_id":0,"_jetpack_memberships_contains_paywalled_content":false,"_jetpack_hide_featured_image":false,"_jetpack_featured_image_duplicate_dismissed":0,"_jetpack_feature_clip_id":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":"","jetpack_post_was_ever_published":false},"categories":[178],"tags":[],"class_list":["post-23287","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-releases"],"acf":[],"jetpack_sharing_enabled":true,"jetpack_featured_media_url":"https:\/\/www.emilysentourage.org\/wp-content\/uploads\/2024-Trailblazer-Award-Announcement.png","post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/23287","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\/1719"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=23287"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/23287\/revisions"}],"predecessor-version":[{"id":23299,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/23287\/revisions\/23299"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media\/23296"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=23287"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=23287"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=23287"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}