Unfortunately, haemophilia A, which is caused by factor VIII deficiency, is about four times as common
as haemophilia B.
After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such
as haemophilia.
Not exact matches
Men inherit one X and one Y chromosome (which encodes the gene for maleness), so they are more likely to express recessive traits from the X, such
as colour blindness and
haemophilia.
As these therapies emerge, dealing with
haemophilia will become less troublesome (see below).
One barrier to
haemophilia therapy is the tendency of factor VIII to prompt the body into producing anti-factor VIII antibodies, known
as inhibitors.
Eloctate, for
haemophilia A, was approved in June and is recommended for an initial infusion once every four days, with a physician adjusting that up to five days or down to three
as appropriate.
We really now have the potential to transform care for people with
haemophilia using a single treatment for people who at the moment must inject themselves
as often
as every other day.
The «transformational» results have particular significance
as the first successful gene therapy trial for the
haemophilia A.