(AP) A study fuels hope that gene therapy can give long-lasting help and perhaps even cure the blood clotting disease called hemophilia.
The disease is caused by lack of a gene that makes a protein needed for blood to clot. Small cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding.
Ten men in the study were given the missing gene through an IV and have been free of nearly all symptoms of the disease for at least a year and now make clotting factor in the normal range.
The therapy is made by Spark Therapeutics and Pfizer. Several other companies are working on similar treatments.
Results were published Wednesday by the New England Journal of Medicine.