Federasyon Mondyal la nan Emofili Rejis Terapi Gene

Federasyon Mondyal la nan Emofili Rejis Terapi Gene
Pwen esansyèl ki soti nan reyinyon 62th ASH Anyèl ak Ekspozisyon an

Federasyon Mondyal la nan Emofili Rejis Terapi Gene

Barbara A. Konkle, MD1,2, Donna sèkèy, MSc3, Mayss Naccache1, Robert Clark4, Lindsey George, MD5, Alfonso Iorio, MD, PhD6, Wolfgang A. Miesbach, MD7, Brian O'Mahony8, Flora Peyvandi9,10, Steven W. Pipe, MD11, Adrian Quartel12, Eileen K Sawyer, PhD13, Mak W Skinner, JD14, Bartholomew J. Tortella, MD, mba15, Crystal Watson, BS16, Leonard A. Valentino, MD17, Ian Winburn18, Johnny Mahlangu19, ak Glenn F. Pierce, MD, PhD20

1Federasyon Mondyal Emofili, Monreyal, QC, Kanada

2Inivèsite Washington, Washington Sant pou maladi senyen, Seattle, WA

3Federasyon Mondyal nan Emofili, Montréal, QC, Kanada

4Sosyete entènasyonal sou tronbozi ak emostaz, Inc, Carrboro

5Lopital Timoun yo nan Philadelphia, Philadelphia, PA

6Inivèsite McMaster, Ontario, Kanada

7Emofili Sant, Inivèsite Lopital Frankfurt, Frankfurt, Almay

8Irish Haemophilia Society, Dublin, Iland

9Angelo Bianchi Bonomi Emofili ak tronbozi Sant, Fondazione IRCCS Ca 'Granda Ospedale majò Policlinico, Milan, Itali

10Angelo Bianchi Bonomi Emofili ak tronbozi Sant, Fondazione IRCCS Ca 'Granda Ospedale majò Policlinico, Milan, Itali

11Pedyatri ak Patoloji, University of Michigan, Ann Arbor, MI

12BioMarin famasetik Inc, Novato

13uniQure Inc, Lexington, MA

14Enstiti pou politik avansman Ltd, Washington, DC

15Spark Therapeutics, Philadelphia, PA

16Ameriken tronbozi ak emostaz Rezo, Decatur, GA

17Fondasyon Nasyonal Emofili (NHF), New York

18Pfizer Inc, New York

19Emofili Comprehensive Care Center, Fakilte nan Syans Sante, University of Witwatersrand la ak NHLS, Johannesburg Nò, Lafrik di sid

20Federasyon Mondyal Emofili, La Jolla, CA

Kle Done Pwen

FASE I / II DESIGN ETID

Apre konsèy nan Ajans Medikaman Ewopeyen an (EMA) ak US Manje ak Administrasyon Dwòg (FDA), WFH GTR SC a te devlope seri sa a debaz nan done yo dwe kolekte sou tout pasyan ki gen emofili, ki moun ki resevwa terapi jèn. Koleksyon done yo pral mande chak trimès pandan premye ane perfusion terapi pòs-jèn ak chak ane apre sa, sou lavi pasyan an. Outreach HTCs te kòmanse nan mwa Out 2020 ak vizit inisyasyon kòmanse nan mwa Oktòb 2020. Rejis la pral ale viv nan mwa janvye 2021.

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