It’s not yet clear how long the effects of this one-time treatment last.
The severity of hemophilia is classified by the amount of factor VIII that the body manages to produce. People with mild disease, who have 5 to 40 percent of typical factor VIII levels, are most likely to have excessive bleeding only after injury or surgery. Moderate hemophilia, with levels 1 to 5 percent of the usual amount, can also include bleeding that occurs spontaneously, without a clear cause.
The standard treatment to prevent bleeding is a replacement infusion of factor VIII, which is needed every 2 to 3 days. That means a person with hemophilia A could face more than 150 of these treatments a year. More recently, a monoclonal antibody that mimics the action of factor VIII has become available, giving adults and children with the disease a once-weekly treatment option.
, which does not cause disease . The virus is taken up by many different cells, but only liver cells can use the instructions to make the clotting protein. It’s also unclear how long the effects of the treatment will last. The researchers report that in 17 participants followed for two years, the clotting protein levels achieved in the first year decreased in the second year. The gene therapy can only be given once because a person would have an immune response to a second dose.
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