Spark Therapeutics's breakthrough hemophilia treatment passes test

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Correcting hemophilia's faulty gene. Ali Gorman reports during Action News at 5 p.m. on August 2, 2017. (WPVI)

A Philadelphia company's breakthrough treatment for hemophilia has passed a big test.

Hemophilia is a condition where the blood doesn't clot like it's supposed to, so people can have severe bleeding even with just a slight injury.

A defective gene causes the problem. But a local research team says they've been able to fix that gene and restore the clotting factor.

In trials, Spark Therapeutics gave three patients infusions of its new treatment.

Correcting the faulty gene restored normal levels of the clotting factor in all of them. And none had either spontaneous bleeds or serious adverse side effects.

Tests on the new compound are continuing with more patients.

Until now, many people with hemophilia have to get infusions of clotting factors several times a week.

Spark's treatment is given just once.
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