Hemophilia is a rare, congenital and hereditary disease caused by a deficiency of certain blood clotting proteins. Those affected have a greater tendency to both spontaneous and traumatic hemorrhages. Severe hemophilia typically occurs early and is diagnosed within the first two years of life. It is a pathology that affects men almost exclusively as the genetic message is associated with the X chromosome.
Despite the important progress made in the treatment of haemophilia, the disease is still related to an important burden for the patient: therapeutic, clinical, physical, psychological and daily activities burden. Gene therapy, on the other hand, has enormous advantages: a single intravenous infusion is enough, the high levels of the clotting factor are sufficient to prevent bleeding, the patient is free from the disease for a long period.
To date, there have been 833 individual therapeutic plans prescribed.
The Medical Clinic Unit 1 is home to the multidisciplinary regional center for the prevention, prophylaxis and advanced treatment of haemophilic arthropathy and the regional center for the prevention, diagnosis and therapy of haemophilias, thrombophilias and complex haemostasis pathologies.
Furthermore, the UOC Clinica Medica 1 is accredited as European haemophilia comprehensive care centres.
Here are the slides from the Press Conference
