In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic
disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served …

Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have
reported remarkable success in ameliorating disease phenotype in hemophilia A and B …

Background Fitusiran, a subcutaneous investigational siRNA therapeutic, targets
antithrombin with the goal of rebalancing haemostasis in people with haemophilia A or …

Inhibitory antibodies (inhibitors) against factor VIII (FVIII) develop in 25% to 35% of
previously untreated patients (PUPs) with severe hemophilia A (SHA). It is the most serious …

Against a background of a rapidly evolving treatment landscape, a contemporary, evidence‐
based consolidated understanding of mortality in people with congenital hemophilia A …

Parkinson's disease (PD) and other chronic and debilitating neurodegenerative diseases
(NDs) impose a substantial medical, emotional, and financial burden on individuals and …

One of the most challenging issues facing us in the treatment of haemophilia is the
development of alloantibodies against infused factor VIII (FVIII) or factor IX (FIX). Inhibitors …

In recent years, the innovation of gene-editing tools such as the CRISPR/Cas9 system
improves the translational gap of treatments mediated by gene therapy. The privileges of …

The classical goals of haemophilia A treatment are to prevent bleeds, minimise the risk of
long-term complications associated with joint damage, and improve quality of life by …

The bleeding disorder hemophilia A (HA) is caused by a single-gene (F8) defect and its
clinical symptom can be substantially improved by a small increase in the plasma …