{"id":20069,"date":"2023-11-21T17:02:46","date_gmt":"2023-11-21T22:02:46","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=20069"},"modified":"2023-12-20T05:15:32","modified_gmt":"2023-12-20T10:15:32","slug":"emilys-entourage-to-host-scientific-symposium-on-the-promise-of-gene-based-therapies-for-cystic-fibrosis","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-to-host-scientific-symposium-on-the-promise-of-gene-based-therapies-for-cystic-fibrosis\/","title":{"rendered":"Emily's Entourage, Kistik Fibrozis i\u00e7in Gen Temelli Tedavilerin Vaadine \u0130li\u015fkin Bilimsel Sempozyuma Ev Sahipli\u011fi Yapacak"},"content":{"rendered":"

Philadelphia, PA - 21 Kas\u0131m<\/strong>, 2023<\/strong> - Nonsense mutasyonu olanlar da dahil olmak \u00fczere kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve tedavi ara\u015ft\u0131rmalar\u0131n\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 kurulu\u015fu olan Emily's Entourage (EE) bug\u00fcn 2023 bilimsel sempozyumunu duyurdu, <\/span>Kistik Fibrozis i\u00e7in Gen Temelli Tedavilerin Vaatleri<\/span><\/i>4 - 5 Aral\u0131k 2023 tarihlerinde Quorum at the University City Science Center'da ger\u00e7ekle\u015ftirilecektir.\u00a0<\/span><\/p>\n

Sempozyumun temas\u0131, KF'de gen temelli tedavilerin geli\u015ftirilmesinde son zamanlarda kaydedilen ilerlemeyi kabul etmekte olup, bu tedaviler KF toplulu\u011funun \u015fu anda mevcut tedavilerden yararlanamayan son 10%'sine ula\u015fma potansiyeline sahiptir ve KF'li herkes i\u00e7in iyile\u015ftirici bir tedaviye yol a\u00e7abilir. \u00d6nemli ilerlemelere ra\u011fmen, KF i\u00e7in yeni gen bazl\u0131 tedavilerin geli\u015ftirilmesi ve sunulmas\u0131nda kritik zorluklar devam etmektedir. Sempozyum, KF bilimsel toplulu\u011fu, end\u00fcstri ve FDA'n\u0131n i\u00e7inden ve d\u0131\u015f\u0131ndan \u00f6nde gelen beyinleri a\u00e7\u0131k bir \u015fekilde payla\u015fmak, i\u015fbirli\u011fi yapmak, zor sorular sormak ve nihayetinde \u00e7\u00f6z\u00fcmler ve stratejik, h\u0131zland\u0131r\u0131lm\u0131\u015f yollar olu\u015fturmak i\u00e7in bir araya getiriyor.<\/span><\/p>\n

Ba\u015fkanl\u0131\u011f\u0131n\u0131 Dr. Patrick Harrison, PhD, Maria Limberis, PhD ve Batsheva Kerem, PhD'nin yapt\u0131\u011f\u0131 ve sadece davetlilerin kat\u0131labildi\u011fi sempozyumda 18 \u00fclkeden 34'\u00fcn \u00fczerinde konu\u015fmac\u0131 ve 86 kat\u0131l\u0131mc\u0131 bir araya geliyor. Sayg\u0131n davetli konu\u015fmac\u0131lar aras\u0131nda 2023 Nobel Fizyoloji veya T\u0131p \u00d6d\u00fcl\u00fc sahibi Dr. Drew Weissman, BS, MS, PhD, MD de yer al\u0131yor.\u00a0<\/span><\/p>\n

Sempozyum iki g\u00fcn boyunca s\u00fcrecek yedi d\u00fc\u015f\u00fcnd\u00fcr\u00fcc\u00fc oturumdan olu\u015fmaktad\u0131r. Her oturumda sunumlar yap\u0131lacak ve derinlemesine tart\u0131\u015fmalar i\u00e7in geni\u015f f\u0131rsatlar sunulacakt\u0131r. B\u00fcy\u00fck final olarak, iki kapan\u0131\u015f oturumu, ustal\u0131kla y\u00f6netilen ve \u00e7ok say\u0131da panelistin g\u00f6r\u00fc\u015fleriyle zenginle\u015ftirilen dinamik yuvarlak masa tart\u0131\u015fmalar\u0131 \u015feklinde olacakt\u0131r.<\/span><\/p>\n

I. <\/b>KF Terap\u00f6tikleri - Ge\u00e7mi\u015f, Bug\u00fcn ve Gelecek<\/b><\/p>\n

Paul Negulescu, PhD | Vertex Pharmaceuticals
\n<\/span>Jennifer Taylor-Cousar, MD, MSCS, ATSF | National Jewish Health
\n<\/span>Kara Foshay, PhD | Kistik Fibrozis Vakf\u0131<\/span><\/p>\n

II. RNA Tabanl\u0131 Yakla\u015f\u0131mlar<\/b><\/p>\n

Drew Weissman, BS, MS, PhD, MD | Pennsylvania \u00dcniversitesi
\n<\/span>Fred Van Goor, PhD | Vertex Pharmaceuticals
\n<\/span>John Lueck, PhD | Rochester \u00dcniversitesi, T\u0131p ve Di\u015f Hekimli\u011fi Fak\u00fcltesi
\n<\/span>Zoya Ignatova, Doktora | Hamburg \u00dcniversitesi<\/span><\/p>\n

III. <\/b>mRNA, tRNA ve Oligon\u00fckleotid Bazl\u0131 Yakla\u015f\u0131mlar i\u00e7in Yeni Da\u011f\u0131t\u0131m Platformlar\u0131ndan Yararlanma<\/b><\/p>\n

Rana Ghosh, PhD | Austin'deki Teksas \u00dcniversitesi
\n<\/span>Alexandra Piotrowski-Daspit, PhD | Michigan \u00dcniversitesi
\n<\/span>Marc Abrams, PhD | Carbon Biosciences<\/span><\/p>\n

IV. KF'de CFTR Gen Tedavisi ve Gen D\u00fczenlemesi<\/b><\/p>\n

Marianne Carlon, PhD | KU Leuven
\n<\/span>Anna Cereseto, Doktora | Trento \u00dcniversitesi
\n<\/span>Smriti Pandey | Harvard \u00dcniversitesi
\n<\/span>Greg Newby, PhD | John Hopkins \u00dcniversitesi
\n<\/span>Ahad Rahim, Doktora | University College London<\/span><\/p>\n

V. Gen Tabanl\u0131 Tedavilerin G\u00fcncel G\u00f6r\u00fcn\u00fcm\u00fc<\/b><\/p>\n

Matt Porteus, MD, PhD | Stanford \u00dcniversitesi
\n<\/span>Kiran Musunuru, MD, PhD, MPH, ML | Pennsylvania \u00dcniversitesi
\n<\/span>Marcus Noyes, PhD | NYU Grossman T\u0131p Fak\u00fcltesi<\/span><\/p>\n

VI. KF i\u00e7in Tedavilerin Geli\u015ftirilmesini H\u0131zland\u0131racak Nadir Gen Tedavilerine \u0130li\u015fkin Perspektif<\/b>\u00a0<\/b><\/p>\n

Maria Limberis, PhD (Moderat\u00f6r)
\n<\/span>David Geller, MD | Arcturus Therapeutics
\n<\/span>Patrick Harrison, Doktora | University College Cork
\n<\/span>Gili Hart, PhD | Splisense
\n<\/span>Craig Hodges, PhD | Case Western Reserve \u00dcniversitesi
\n<\/span>Prateek Shukla, MD | FDA
\n<\/span>Richard Horgan, MBA | Cure Rare Disease<\/span><\/p>\n

VII. KF Merce\u011finden Gen Tedavisi Klinik Ara\u015ft\u0131rmalar\u0131ndaki Zorluklar<\/b><\/p>\n

Jennifer Taylor-Cousar, MD, MSCS, ATSF | National Jewish Health (Moderat\u00f6r)
\n<\/span>Marty Burke, PhD, MD | Illinois \u00dcniversitesi
\n<\/span>Ilan Ganot, MBA | Solid Biosciences
\n<\/span>Jessica Lee | Spirovant
\n<\/span>Joel Schneider, PhD | Carbon Biosciences
\n<\/span>Wolfgang Seibold, MD | Boehringer Ingelheim
\n<\/span>Alan Cohen, MD | 4DMT<\/span><\/p>\n

\"Son y\u0131llarda gen bazl\u0131 tedavilerin geli\u015ftirilmesinde muazzam bir ilerleme kaydedilmi\u015ftir. Emily's Entourage'\u0131n Ba\u015f Bilimsel Sorumlusu Chandra Ghose, KF toplulu\u011fundaki herkes ve \u00f6zellikle de mevcut mutasyon hedefli tedavilerden fayda g\u00f6rmeyen ve hala umutsuzca tedavi se\u00e7enekleri bekleyen son 10%'deki ki\u015filer i\u00e7in bir umut \u0131\u015f\u0131\u011f\u0131 temsil etti\u011fi i\u00e7in bu ilerlemeden cesaret al\u0131yoruz\" dedi. \"KF toplumunun 100%'sinin genetik mutasyonlar\u0131ndan ba\u011f\u0131ms\u0131z olarak uzun ve sa\u011fl\u0131kl\u0131 bir ya\u015fam s\u00fcrmelerini sa\u011flayan hayat kurtar\u0131c\u0131 tedavilere sahip oldu\u011fu bir gelece\u011fe do\u011fru h\u0131zl\u0131 bir yolu i\u015fbirli\u011fi i\u00e7inde \u00e7izmek i\u00e7in sempozyumumuzda birinci s\u0131n\u0131f liderleri bir araya getirmekten heyecan duyuyoruz.\"<\/span><\/p>\n

EE, 2023 sempozyumu Sunum, R\u00fcya Tak\u0131m ve Menek\u015fe Seviyesi sponsorlar\u0131na te\u015fekk\u00fcr eder, <\/span>Viatris,<\/b> 4D Molek\u00fcler Terap\u00f6tikler<\/b>, <\/span>Vertex Pharmaceuticals, Inc., ReCode Therapeutics, Boomer Esiason Vakf\u0131,<\/b> ve daha fazlas\u0131<\/span> Bu kritik etkinli\u011fi ve sa\u011flad\u0131\u011f\u0131 ilerlemeyi m\u00fcmk\u00fcn k\u0131lan destekleri i\u00e7in te\u015fekk\u00fcr ederiz.\u00a0\u00a0<\/span><\/p>\n

##<\/span><\/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 kay\u0131t defteri ve klinik deney e\u015fle\u015ftirme program\u0131 geli\u015ftirmi\u015f ve y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirmeye y\u00f6nelik d\u00fcnya \u00e7ap\u0131nda \u00e7abalara \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 <\/span>emilysentourage.org<\/span><\/a>.\u00a0<\/span><\/p>\n

 <\/p>\n

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

Emily'nin \u00c7evresi<\/span><\/p>\n

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

Philadelphia, PA - 21 Kas\u0131m 2023 - Nonsense mutasyonlar\u0131 olanlar da dahil olmak \u00fczere kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve bir tedavi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 olan Emily's Entourage (EE), bug\u00fcn 2023'te d\u00fczenlenecek olan Kistik Fibrozis i\u00e7in Gene Dayal\u0131 Tedavilerin Vaadi adl\u0131 bilimsel sempozyumunu duyurdu. <\/p>\n

<\/div>\n

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