Haemophilia A and B are rare congenital, recessive X-linked disorders caused by lack or
deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease …

Circular RNAs (circRNA) represent a distinct class of covalently closed-loop RNA molecules,
which play diverse roles in regulating biological processes and disease states. The …

In the last decade, enormous progress has been made in the development of gene therapy
for hemophilia A and B. After the first encouraging results of intravenously administered …

Congenital haemophilia A (factor VIII deficiency) and B (factor IX deficiency) are X-linked
bleeding disorders. Replacement therapy has been the cornerstone of the management of …

Results from the main parts (24 weeks) of 2 concizumab phase 2 trials are presented:
explorer4 in hemophilia A (HA) or B (HB) with inhibitors (HAwI/HBwI) and explorer5 in HA …

Hemophilia A (HA) and hemophilia B (HB) are the most common severe bleeding disorders.
Replacement therapy, providing the missing coagulation factor, has been the mainstay of …

Abstract Background The Hemophilia Joint Health Score (HJHS) was developed and
validated to detect arthropathy in children. Additional evidence is required to show validity in …

Background Factor VIII replacement products have improved the care of patients with
hemophilia A, but the short half-life of these products affects the patients' quality of life. The …

Haemophilia is a rare disease for which the approved therapeutic options have remained
virtually unchanged for 50 years. In the past decade, however, there has been an explosion …

Haemophilia A and haemophilia B are congenital X-linked bleeding disorders caused by
deficiency of coagulation factor VIII (FVIII) and IX (FIX), respectively. The preferred treatment …