Since females have two X chromosomes (XX), a healthy gene on one can usually compensate for a faulty one on the other. The Royal Connection
There are two primary forms, depending on which protein is missing:
Decades ago, the prognosis for someone with "bleeder's disease" was poor, often involving a short life expectancy and severe physical disability. Today, the landscape has changed: bleeder's disease
Also known as "Christmas Disease" (named after the first patient diagnosed with it), caused by a lack of Factor IX .
Recent breakthroughs are aiming for "cures" by using viruses to deliver functional genes into the patient's liver, allowing the body to produce its own clotting factors. Since females have two X chromosomes (XX), a
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Since males have only one X chromosome (XY), a single faulty gene causes the disease. Recent breakthroughs are aiming for "cures" by using
Regular infusions of replacement clotting factors allow patients to lead near-normal lives.