Practice Points
Ensuring the Safety of Medicines for Haemophilia
A medicine’s journey from laboratory bench to bedside is a highly regulated process – one that takes years. Trials involve thousands of people who take a compound that may or may not become a medicine and for which the risks are largely unknown. The safety of volunteers and patients who participate in these trials is …
EHL factor concentrates
Haemophilia is an X-linked inherited bleeding disorder characterised by reduced levels of Factor VIII or IX levels (haemophilia A and B) respectively [Shrivastava et al, 2013]. In its severe form (FVIII or IX <0.01IU/dl) haemophilia results in recurrent joint and muscle bleeds that predispose to arthropathy, muscle contracture and disability. Prophylaxis with clotting factor replacement …