Bringing Gene Therapy Into Real World Clinical Practice
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach +2 more
wiley +1 more source
Replicative Level of Hepatitis C Virus(HCV) in Chronic Hepatitis C.
HAYASHI, Norio, KAMADA, Takenobu
openaire +3 more sources
Beyond Standard Half‐Life: Real‐world Pharmacokinetics of Efanesoctocog Alfa in a Single Centre
ABSTRACT Introduction Efanesoctocog alfa (EFA) is an ultra–extended half‐life factor VIII (FVIII) developed to address limitations of conventional prophylaxis in haemophilia A. Although the XTEND trials reported low interindividual pharmacokinetic (PK) variability, real‐world data remain important to better characterize PK profiles across patient ...
Laurent Sattler +6 more
wiley +1 more source
Hepatitis C Virus (HCV)-Mediated Activation of Hexokinase Domain-Containing Protein 1 (HKDC1) Promotes Hexokinase Activity and Metabolic Reprogramming. [PDF]
Fiadjoe HK +3 more
europepmc +1 more source
Hepatitis C virus (HCV) infection in Malaysia: findings from a nationwide cross-sectional study. [PDF]
Muhammad EN +6 more
europepmc +1 more source
UK Infected Blood Inquiry–An Historical Appraisal
ABSTRACT Introduction This appraisal considers the 2018 UK Infected Blood Inquiry and its Report (2024) in the context of the history of haemophilia treatment developments over the past fifty‐five years, and the tragic impacts of the AIDS pandemic. Aim The paper appraises the conduct and findings of the IBI and its Report in relation to HIV and AIDS ...
Christopher A. Ludlam
wiley +1 more source
Characterization of near-complete human <i>Pegivirus</i> 2 (HPgV-2) genomes in individuals co-infected with hepatitis C virus (HCV) in Cameroon. [PDF]
Mounchili-Njifon A +13 more
europepmc +1 more source
Cytokine profile and viral diversity in the early seronegative stage of community-acquired hepatitis C virus (HCV) infection. [PDF]
Radkowski M +8 more
europepmc +1 more source

