With longer follow-up out to one year, bleeding episodes have been almost completely eliminated for a small group of hemophilia B patients infused once with an experimental gene therapy from Spark Therapeutics (ONCE).
The updated results from Spark’s Phase 1/2 clinical trial, published Wednesday night in the New England Journal of Medicine, provide further evidence to support a groundbreaking change in the way the inherited blood-clotting disorder is treated — and potentially cured.
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