Meshari Al-Armally lives in Kuwait and has haemophilia A

DISEASE AREAS

Haemophilia

Discovering safe and effective treatments for people living with haemophilia and other rare bleeding disorders

Haemophilia

Haemophilia is a rare and serious X-chromosome linked congenital bleeding disorder that affects the blood's ability to clot, meaning that people with haemophilia bleed for a longer time than normal. It is estimated that about 1 in 10,000 people are affected by haemophilia, with 450,000 people living with haemophilia worldwide.

Haemophilia 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 haemophilia, each associated with deficiency of a particular coagulation factor.

The most common type is haemophilia A, where the person does not have enough coagulation factor VIII (FVIII).

Haemophilia B is less common, with people not having enough coagulation factor IX (FIX), representing only 15–20% of total haemophilia 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.

Carl Lyons from Denmark living with haemophilia A

Living with haemophilia

Different varieties of haemophilia are associated with several types of coagulation factors. There are different treatments depending on the missing factor, and currently, all are administered by infusion therapy injected into a vein.

This replacement therapy can be given to combat a bleeding episode that's in progress. Or it can also be administered at home on a regular schedule to help prevent bleeding episodes. For some people living with haemophilia, they must receive continuous replacement therapy.

Meet Carl Lyons in the video above and learn about life as a boy with severe haemophilia A.

Life with haemophilia

450k

people have haemophilia globally.

59%

 have limited mobility.

94%

feel supported by their partners.

51%

of parents feel their son’s haemophilia makes the whole family closer.

Meet Ulla Hedner, leading haemophilia researcher

Working towards the next big breakthrough

Severe haemophilia usually becomes apparent in the first years of life - often when the child starts to move about independently. Haemorrhages often occur in the joints, particularly the weight bearing joints such as knees and ankles.

This pain is often unbearable, and helping young children and adults living with haemophilia to avoid this drives the passion in our research.

We combine our experience in protein design with collaborations in the global scientific community to discover and develop effective and safe medicines for people with haemophilia and other rare bleeding disorders.

In the video above, meet Ulla Hedner, a leading pioneer in haemophilia treatment. Ulla explains her passion and dedication to help haemophilia patients.

Haemophilia illustration displaying lab equipment.

Advancing treatments in haemophilia and in rare blood disorders

Our scientists are exploring innovative long-acting and subcutaneous treatment solutions for haemophilia 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 haemophilia 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 haemophilia
  • 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.

Explore our ambitious R&D pipeline.

Our rare diseases science hub

Explore our searchable database of scientific resources on rare bleeding disorders. You can find all recent journal publications and a variety of educational resources.