The hospital has demonstrated the therapy's efficacy in substantially elevating the deficient clotting factor with just a single dose.
This increase enables patients to engage in daily activities without the constant requirement for preventive treatment. This advancement heralds a transformative improvement in the quality of life for patients with haemophilia.
Gene therapy for haemophilia works by introducing new, functional genes into the patient's body, enabling it to independently manufacture the previously deficient clotting factor.
Essentially, these new genes instruct the patient's own cells on how to synthesiae the blood clotting factor. This process is typically achieved with just one therapeutic dose delivered intravenously.
Haemophilia arises from a genetic mutation that prevents the body from producing a necessary clotting factor. Patients with this condition typically require lifelong management, which involves regular injections of clotting factor directly into the bloodstream every 3–5 days to prevent and control bleeding episodes.
This disorder causes significant bleeding in various body parts, particularly the joints, which can lead to symptoms such as swelling, pain, and limited joint mobility. It can also cause bleeding in the muscles and potentially dangerous internal bleeding within the brain.
This achievement is a testament to the KFSH&RC's commitment to leveraging every available resource and integrating cutting-edge technologies to improve treatment outcomes, patient experiences and the efficiency of healthcare delivery, striving to be the preferred provider for specialised healthcare needs.