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.

หนังสือแฮโมฟิเลียภาพ Lisa มิลเลอร์จากลอนดอน (แม่) และไอแซค (7) กับน้องสาว Lauryn (5) - Isaac มี haamophilia

เชื่อมโยงสองบรรทัดชื่นชมอย่างมากmarkwaugh.Net

Isaac Miller lives in the UK and has haemophilia A.

Living with haemophilia

In severe cases of haemophilia, 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 haemophilia are still undiagnosed or inadequately treated.

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.

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.