Haitian Creole
English
Afrikaans
Albanian
Arabic
Armenian
Azerbaijani
Basque
Belarusian
Bulgarian
Catalan
Chinese (Simplified)
Chinese (Traditional)
Croatian
Czech
Danish
Dutch
Estonian
Filipino
Finnish
French
Galician
Georgian
German
Greek
Haitian Creole
Hebrew
Hindi
Hungarian
Icelandic
Indonesian
Irish
Italian
Japanese
Korean
Latvian
Lithuanian
Macedonian
Malay
Maltese
Norwegian
Persian
Polish
Portuguese
Romanian
Russian
Serbian
Slovak
Slovenian
Spanish
Swahili
Swedish
Thai
Turkish
Ukrainian
Urdu
Vietnamese
Welsh
Yiddish
Pwen esansyèl Soti nan 30yèm Kongrè ISTH la
Rezilta B-LIEVE, yon etid Faz 1/2 Konfimasyon Dòz FLT180a AAV Gene Therapy nan Pasyan ki gen emofili B.
Prezante pa: Guy Young, MD, Keck School of Medicine nan USC, Los Angeles, Kalifòni, Etazini.
G. Young1, P. Chowdary2, S. Barton3, D. Yee3, F. Ferrante3
1Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA, Los Angeles, Kalifòni, Etazini.
2Royal Free Hospital, Lond, Angletè, Wayòm Ini
3Freeline, Stevenage, Angletè, Wayòm Ini
Kle Done Pwen
Nivo FIX pou 3 premye pasyan ki gen emofili B ki te resevwa dòz FLT180a AAV nan etid B-LIEVE faz 1/2. Valè FIX yo soti nan yon tès yon etap nan yon laboratwa santral; liy orizontal tirè se limit ki pi ba nan ekspresyon nòmal FIX (50 IU/dL). Apati 23 me 2022, done koupe, nivo FIX yo te 93, 92, ak 80 nan jou etid 77, 56, ak 36 pou pasyan 1, 2, ak 3, respektivman.
FIX (wouj) ak ALT (jòn) nivo pou 3 pasyan yo pran dòz FLT180a nan etid B-LIEVE. Twa semèn apre perfusion FLT180a, pasyan yo te kòmanse yon kou ki diminye nan kortikoterapi oral ak yon kou kout nan oral tacrolimus kòm pwofilaksi pou minimize repons iminitè ki gen rapò ak vektè. Liy pwenti vètikal yo se perfusion FIX ekzojèn pwofilaktik yo resevwa nan jou apre perfusion FLT180a.
RELATED kontni
