Sèvi ak Rejis World Federation of Haemophilia Gene Therapy pou Suivi alontèm Pasyan Haemophilia Trete ak Gene Therapy

Pwen esansyèl Soti nan Kongrè Mondyal WFH 2022 la

Sèvi ak Rejis World Federation of Haemophilia Gene Therapy pou Suivi alontèm Pasyan Haemophilia Trete ak Gene Therapy

Prezante pa: Barbara A. Konkle, MD, University of Washington, Seattle, Washington, USA

Barbara A. Konkle¹, Donna sèkèy², Mayss Naccache², Cary Clark³, Lindsey A. George⁴, Alfonso Iorio⁵, Wolfgang Miesbach⁶, Declan Noone⁷, Flora Peyvandi⁸, Steven Pipe⁹, Michael Recht¹⁰, Mak Skinner¹¹, Leonard Valentino¹², Johnny N. Mahlangu¹³, Glenn F. Pierce²

¹University of Washington, Seattle, Etazini
²Federasyon Mondyal Emofili, Monreyal, Kanada
³Sosyete Entènasyonal sou tronboz ak emostaz, Carrboro, Etazini
⁴The Children's Hospital of Philadelphia, Philadelphia, Etazini
⁵Depatman Metòd Rechèch Sante, Prèv, ak Enpak, McMaster University, Hamilton, Kanada
⁶Klinik Medikal 2/Enstiti Medsin Transfizyon Inivèsite Lopital Frankfurt, Almay
⁷Ewopeyen Haemophilia Consortium, Bèljik
⁸Università degli Studi di Milano, Itali
⁹Depatman Pedyatri ak Patoloji, University of Michigan, Ann Arbor, Etazini
¹⁰American Thrombosis and Hemostasis Network, Rochester, Etazini
¹¹Institutefor Policy Advancement Ltd., ak Depatman Rechèch Metòd, Prèv, ak Enpak, McMaster University, Washington, District of Columbia
¹²Fondasyon Nasyonal Emofili, New York, Etazini
¹³Inivèsite Witwatersrand, Lafrik di sid

Rejis terapi jèn WFH (GTR) se yon rejis potansyèl, obsèvasyon, ak longitudinal ak yon objektif jeneral nan kolekte done pou tout pasyan ki resevwa terapi jèn pou emofili, tou de nan esè klinik ak nan yon faz apre maketing si ak lè yo apwouve. Sa yo se objektif prensipal ak segondè nan rejis la.

Pou kapab aplike GTR a, WFH ap rive jwenn sant tretman emofili endividyèl (HTCs), kòmanse ak moun ki enplike nan esè klinik terapi jèn emofili. Y ap mande rejis nasyonal emofili pou kontribiye tou. WFH ap travay tou ak endistri pou devlope yon pwosesis pou jwenn done ki sòti nan esè klinik yo fini.