Loma Linda University Health Launched Groundbreaking HEMGENIX Gene Therapy for Hemophilia
Loma Linda University Health's Centre provides HEMGENIX, the first gene therapy approved by the FDA for haemophilia B, along with next-generation gene therapy for haemophilia A. This advancement has the potential to significantly improve the lives of many children and adults affected by these conditions.
Haemophilia is a genetic disorder that impacts the blood's ability to clot, posing a serious health concern for individuals with the condition.Even minor injuries can lead to severe health complications due to uncontrolled bleeding.
Haemophilia is typically inherited through the X chromosome, which explains why it mainly affects males, while females are often carriers.
Traditionally, treating haemophilia required patients to receive frequent infusions of clotting factors, necessitating multiple self-injections each week to prevent dangerous bleeding episodes. This treatment can be both painful and limiting.
HEMGENIX allows patients to produce their own missing clotting factors. This single treatment consists of a three-hour infusion that delivers a modified gene to the liver, enabling the body to naturally produce the necessary clotting factors.
Patients receiving HEMGENIX can experience a significant increase in clotting factor levels, from below 1% to approximately 30-40%. Although the normal range starts at 55%.
This improvement can greatly reduce the frequency and severity of bleeding episodes, enabling patients to enjoy activities like walking and playing sports without the constant worry of life-threatening bleeding.
