Bringing us closer to ‘once-in-a-lifetime’ hemophilia treatment with experimental gene therapies

gene therapy

Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.

After trying for decades to develop a gene therapy to treat this disease, researchers are starting to succeed. In recent experiments, brief intravenous infusions of powerful new treatments have rid patients — for now, at least — of a condition that has shadowed them all their lives.

For now, “we are anticipating that this is a once-in-a-lifetime treatment,” said Dr. Steven Pipe.

People with hemophilia often are taught as children to avoid most sports and to find professions that will not require much physical activity. Many move to cities to gain easier access to treatment.

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They may change jobs to get insurance needed to cover medical bills for hospitalizations and surgeries that can reach $1 million a year, plus an average of $250,000 to $300,000 a year for the clotting proteins. (The shots alone can cost as much as $1 million per year.)

Despite their vigilance, most with severe disease eventually develop permanent joint damage from bleeds, often leading to surgery for ankle fusion or hip or knee replacements at an early age. Most live with chronic pain from past bleeds.

Now, though, researchers see the start of a new era.

Read full, original post: They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong.

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