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 …

Factor VIII (FVIII) replacement therapy is the foundation of treatment in hemophilia A and is
effective unless a patient develops an alloantibody (inhibitor) against exogenous FVIII …

Background: The large observed variability in hemophilia prevalence prevents robust
estimation of burden of disease. Objective: To estimate the prevalence and prevalence at …

Background Emicizumab, a subcutaneously administered, humanised, bispecific,
monoclonal antibody, is approved to treat people with haemophilia A of all ages with and …

Background Mutations in LRRK2, the gene that encodes leucine-rich repeat kinase 2, are a
cause of Parkinson's disease (PD). The International LRRK2 Consortium was established to …

The objectives of this paper were to study the reported haemophilia A prevalence (per 100
000 males) on a country‐by‐country basis and address the following: Does the reported …

Many human Y-chromosomal deletions are thought to severely impair reproductive fitness,
which precludes their transmission to the next generation and thus ensures their rarity in the …

Although persons with hemophilia are known to be at increased risk of death, no studies
have examined the source of medical care and other personal characteristics for …

I estimate per nucleotide rates of spontaneous mutations of different kinds in humans directly
from the data on per locus mutation rates and on sequences of de novo nonsense …

Chronic joint disease from repeated bleeding into joints is a serious complication of
hemophilia. To measure the extent of and to identify risk factors for deviations from normal in …