Disclaimer statement
Warning!
Hemophilia
Hemophilia is a rare and serious X-chromosome linked congenital bleeding disorder that affects the blood's ability to clot, meaning that people with hemophilia bleed for a longer time than normal. It is estimated that about 1 in 10,000 people are affected by hemophilia, with 450,000 people living with hemophilia worldwide.
Hemophilia is characterised by deficiencies of coagulation factors, and is typically passed down from parent to child, although about a third of cases are caused by a spontaneous mutation. There are two different types of hemophilia, each associated with deficiency of a particular coagulation factor.
The most common type is hemophilia A, where the person does not have enough coagulation factor VIII (FVIII).
Hemophilia B is less common, with people not having enough coagulation factor IX (FIX), representing only 15–20% of total hemophilia cases.
The genes for both coagulation factors are on the X chromosome, which is why it mainly affects males who inherit an affected maternal X chromosome.
Driving change for a life without limitations
Hemophilia is a lifelong condition that has a significant impact on the lives of individuals and their families. Fortunately, recent innovations have changed the treatment landscape for hemophilia and other rare bleeding disorders dramatically.
We are committed to driving change for a future where everyone with hemophilia and other rare bleeding disorders can get the treatment they need and live a life with as few limitations as possible.
Isaac Miller lives in the UK and has hemophilia A.
Living with hemophilia
In severe cases of hemophilia, continuous bleeding occurs after minor trauma or even in the absence of injury (known as spontaneous bleeding). Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. This can lead to chronic pain and limited mobility.
Medication is given as injections, either when a bleed occurs (on demand therapy), or on a regular basis to prevent bleeds from occurring in the first place.
Many people with hemophilia are still undiagnosed or inadequately treated.
Life with hemophilia
Life with hemophilia
450k
people have hemophilia globally.
59%
have limited mobility.1
94%
feel supported by their partners.
51%
of parents feel their son’s hemophilia brings the whole family closer.
Advancing treatments in hemophilia and in rare blood disorders
Our scientists are exploring innovative long-acting and subcutaneous treatment solutions for hemophilia and rare blood disorders. These solutions are aimed at reducing the current treatment burden and improving clinical outcomes, and will be complemented by research into oral treatments and gene therapy.
We strive for offering better quality of life to people living with hemophilia and other rare bleeding disorders.
We often work in partnerships to discover novel targets and innovative compounds and technologies that address unmet medical needs. Our focus is on:
- Novel prophylactic agents in hemophilia
- Cell and gene-based therapy approaches
- Clinical assets in rare blood disorders such as sickle-cell disease
Today, our pipeline has exciting potential to ensure we are at the forefront of finding the next generation of innovative medicines.
