Recently, U.S. drugmaker Pfizer Inc said that its experimental gene therapy for the treatment of haemophilia B, met its main goal in a late-stage study.

 

Details

Data from the study showed that a single dose of the therapy was superior to the current standard of care in helping reduce the bleeding rate in patients with moderately severe to severe forms of hemophilia B.

 

What is Haemophilia?

  • It is a medical condition, mostly inherited, in which the ability of blood to clot is severely reduced.
  • This disease is caused by a mutation in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot.
  • This change or mutation can prevent the clotting protein from working properly or to be missing altogether. These genes are located on the X chromosome.
  • Men are more vulnerable to haemophilia than women. It is a rare disease where in about 1 in 10,000 people are born with it.