Open Access<p>The Patient Experience of Gene Therapy for Hemophilia: Qualitative Interviews with Trial Patients</p>
Author(s) -
Wolfgang Miesbach,
Robert Klamroth
Publication year - 2020
Publication title -
patient preference and adherence
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.885
H-Index - 48
ISSN - 1177-889X
DOI - 10.2147/ppa.s239810
Subject(s) - haemophilia , medicine , factor ix , anxiety , genetic enhancement , haemophilia b , clinical trial , physical therapy , haemophilia a , pediatrics , psychiatry , gene , biochemistry , chemistry
The phase ½ hemophilia B clinical trial (AMT-060) demonstrated stable endogenous FIX levels after 3.5 years (mean FIX activity between 5.1% and 7.5%) with continued reductions in annualized bleeds to near zero with the higher dose, and a 78-96% reduction by year in exogenous FIX use.