The man's brother was almost bedridden
by haemophilia, but the soldier's symptoms were so mild that he did not even realize that he had the disease until he was shot on the battlefield.
Biopharmaceutical treatments, led
by haemophilia, make up around 20 percent of Novo's sales, with diabetes and obesity products accounting for the remaining 80 percent.
Not exact matches
Existing
haemophilia treatments tip the balance towards clotting
by adding what the body lacks — the clotting factor that is missing or defective.
Indeed, exposure of the protein produced
by the nanoparticle - based gene therapy to the gut mucosa prevents inhibitor development and restores clotting - factor activity in mouse models of both
haemophilia A and B. «This approach really could hold big benefit for patients,» says Jörg Schüttrumpf, a transfusion - medicine specialist who led one of the studies performed at the German Red Cross Blood Donor Service in Frankfurt.
Depending on the person, the amount of factor VIII — the protein missing in
haemophilia A — in the bloodstream drops
by half in a mere 8 — 12 hours.
Unfortunately,
haemophilia A, which is caused
by factor VIII deficiency, is about four times as common as
haemophilia B.
Disorders like
haemophilia that are caused
by faults on the X chromosome are therefore more common in males.
For all the advantages of these extended - life molecules, the researchers predict that they will be supplanted in perhaps a decade
by advances in gene therapy, which will enable people with
haemophilia to produce their own clotting factors.
Replacing the clotting ability lacking in
haemophilia has been the treatment since the 1840s, when attempts were made to treat people with the disease
by transfusion with whole blood from people with normal clotting.
A «cure» for
haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led
by the NHS in London.
Almost exactly a year ago, we reported on a gene therapy for
haemophilia that was in development
by the Children's Hospital of Philadelphia (and subsequently the CHOP spinout Spark Therapeutics).
The therapy hoped to resolve some of the problems surrounding
haemophilia treatment
by exchanging the continual infusions for a single vector infusion that could permanently boost Factor IX expression.
Some concerns had been raised that
by infusing patients with such a high activity gene, the treatment would overcorrect for
haemophilia and give rise to thrombosis.
Another noteworthy issue is that psychological or psychiatric conditions are reported
by 47 % of PWH, with 29 % relating these symptoms to
haemophilia.4 This is even more relevant considering that psychological factors can influence both pain experience and QoL in PWH.12 Interestingly, Cassis et al 6 state that variations in QoL are better explained
by psychosocial, rather than clinical predictors.