Successes with experimental gene therapies suggest that hemophilia - the inability to form blood clots which leads to uncontrolled bleeding and chronic pain - may be curable.
The New York Times reports scientists are “edging closer” to working gene therapies, which include injections, that have rid hemophilia patients of the condition.
“We are anticipating that this is a once-in-a-lifetime treatment,” Dr. Steven Pipe, director of the hemophilia and coagulation disorders program at the University of Michigan, told The Times’s Gina Kolata.
Several biotech companies are now rushing to get their gene therapies on the market.
According to The Times, there are 20,000 hemophilia patients in the United States who lack one of two proteins needed for blood to clot.
The Mayo Clinic says there are several types of hemophilia, and most forms are inherited. However, about 30 percent of people with hemophilia have no family history of the disorder.
The clinic explains that for hemophiliacs, small cuts usually aren't much of a problem. The greater health concern is deep bleeding inside the body, especially in the knees, ankles and elbows.
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