Mannucci Pier Mannuccio
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi, Milano.
Recenti Prog Med. 2023 May;114(5):247-249. doi: 10.1701/4032.40071.
In Italy have materialized changes pertaining to the striking progress for replacement and non-replacement therapies in patients with hemophilia A and B. This editorial looks at further progress that is expected to occur in Italy as elsewhere with the approval of two gene therapies for the hemophilias, as well as the forthcoming approval and availability of a new factor VIII product with a very extended plasma half-life that can be administered no more frequently than once weekly.
在意大利,血友病A和B患者替代和非替代疗法取得了显著进展,相关变化已经显现。随着两种血友病基因疗法获批,以及一种新型凝血因子VIII产品即将获批并上市,其血浆半衰期极长,给药频率不超过每周一次,本社论探讨了预计在意大利以及其他地方将会取得的进一步进展。