Hemophilia is an inherited disorder caused by low or deficient level of clotting factor/s leading to the inability to form proper blood clot. This deficiency is caused by genetic mutations on the X chromosome encoding both clotting factors VIII (FVIII) and IX (FIX).
There are two main kinds of Hemophilia that depend on the type of missing factor:
Approximately % 50
of people with Hemophilia around the world still receive inadequate treatment or have no access to treatment
More than $ 199950
treatment costs encompasses per patient per year, including indirect costs
Approximately % 1
of patients with severe form of Hemophilia will develop inhibitors against injected factors
Rare inherited disorder (genetic mutations on the X chromosome)
2 mains types : hemophilia A (factor VIII) and B (factor IX)
Low or deficient levels of clotting factors
3 forms depending on clotting factor level : severe, moderate, mild
One injection of factors every 2-3 days= prophylactic treatment
Spontaneous bleeding is the most dangerous complication
The goal of Defymed is to use MailPan® technology as a platform for cell encapsulation. In the case of Hemophilia A, the aim is to macro-encapsulate factor VIII secreting cells in immuno-protective membrane.
The combined solution implies and offers: