Enpak transfè jèn Valoctocogene Roxaparvovec pou emofili A grav sou kalite lavi ki gen rapò ak sante
Pwen esansyèl nan 15yèm Kongrè Anyèl EAHAD la

Enpak transfè jèn Valoctocogene Roxaparvovec pou emofili A grav sou kalite lavi ki gen rapò ak sante

B.O'Mahony1,2; J.Mahlangu3; K.Peerlinck4; JDWang5; G.Lowe6; CWTan7; A.Giermasz8; H.Tran9; TLKhoo10; E.Cockrell11; D.Pepperell12; H.Chambost13; MLFernández14; R.Kazmi15; E.Majerus16; MWSkinner17,18; R.Klamroth19; J.Quinn20; H.Yu20; WYWong20; A.Lawal20; TMRobinson20; B.Kim20

1Ilandè Haemophilia Society;2Trinity College, Dublin, Iland;3 Emophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand ak NHLS, Johannesburg, Lafrik di sid;4 Depatman Medsin vaskilè ak emostaz ak sant emofili, Lopital Inivèsite Leuven, Leuven, Bèljik;5 Centerfor Maladi Ra ak Emofili, Taichung Veterans General Hospital, Taichung, Taiwan, Pwovens Lachin;6 West Midlands Comprehensive Care Haemophilia Centre, Queen Elizabeth Hospital, Birmingham, Wayòm Ini;7 Depatman ematoloji, Royal Adelaide Hospital, Adelaide, Ostrali;8 Sant Tretman Emofili, University of California Davis, Sacramento, Etazini;9 Inite emostaz ak tronboz, Sant Tretman Haemophilia, Lopital Alfred, Melbourne;10 Enstiti ematoloji, Royal Prince Alfred Hospital, Camperdown, Ostrali;11 Pedyat Hematology Oncology, Saint Joseph's Children's Hospital, Tampa, Etazini;12 Depatman Ematoloji, Lopital Fiona Stanley, Murdoch, Ostrali;13 AP-HM, Depatman Pedyat Ematoloji Onkoloji, Timoun Lopital La Timone, Aix Marseille University, INSERM, INRA, C2VN, Marseille, Frans;14 Complejo Hospitalario Universitario A Coruña, A Coruña, Espay;15 Depatman ematoloji, Southampton University Hospital, Southampton, Wayòm Ini;16 Depatman Medsin, Washington University nan St. Louis, St.17 Institute for Policy Advancement Ltd, Washington, Etazini;18 McMaster University, Hamilton, Kanada;19 Sant Tretman Emofili Swen konplè, Vivantes Klinikumim Friedrichshain, Bèlen, Almay;20 BioMarin famasetik Inc, Novato, Etazini

Kle Done Pwen

Haemo-QOL-A Total ak Nòt Domèn

aP <0.0001, bP <0.01, cP <0.05, ak dP <0.001. Boldface endike ke CFB te gen yon eksplorasyon P-valè <0.05. Haemo-QOL-A, Kesyonè Kalite Vi pou Haemophilia-espesifik pou Adilt.

Rezilta analiz Haemo-QOL-A pou faz 3 GENEr8-1 esè valoctocogene roxaparvovec terapi jèn pou emofili A. Yo montre domèn ak nòt total pou debaz, semèn 26, ak semèn 52. Woz lonbraj endike yon chanjman mwayèn nan debaz. (CFB) ki se ≥ diferans klinik enpòtan (CID) pou nòt domèn (6.0) ak nòt total (5.5).

RELATED kontni

Webinars entèaktif
Imaj

Please enable the javascript to submit this form

Sipòte pa sibvansyon edikasyon bay Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, ak uniQure, Inc.

Esansyèl SSL