The King Faisal Specialist Hospital and Research Centre (KFSH&RC) has successfully administered an advanced gene therapy for eight patients suffering from hemophilia, a genetic bleeding disorder. 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 hemophilia.
Gene therapy for hemophilia 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 synthesize the blood clotting factor. This process is typically achieved with just one therapeutic dose delivered intravenously.
Hemophilia 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 three to five days to prevent and control bleeding episodes.
Hemophilia is the result of a genetic mutation that affects the genes responsible for instructing the production of a specific blood protein — the clotting factor. This protein is crucial for the blood clotting process that stops bleeding. The mutation results in an inability to produce the necessary clotting factor, which is what leads to the bleeding disorder characteristic of hemophilia.
This disorder causes significant bleeding in various body parts, particularly the joints, which can lead to symptoms like 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 specialized healthcare needs.
The King Faisal Specialist Hospital and Research Center is globally recognized for its exceptional specialized healthcare services and groundbreaking innovations. It stands as an advanced hub for medical research and education, committed to advancing medical technologies and raising the standard of healthcare on a global scale. Through collaborations with prominent local, regional, and international institutions, it aspires to deliver world-class services in clinical care, research, and education.