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Hemophilia A gene therapy shows promise in phase 3 clinical trial

 Last Update: 2022-04-25
 Source: Internet
 Author: User

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New England Journal of Medicine (2022)

Research by an international team of researchers has found more evidence that a new gene therapy for hemophilia A may help some people, in their paper published in the New England Journal of Medicine by a group describing the characteristics of clinical trials and provide data on the effectiveness of the treatment

Hemophilia A is a genetic disorder that affects the production of a protein called factor 8, and people with the disease experience blood clotting problems

Currently, hemophilia A patients are treated with factor VIII infusions, and while it is effective in allowing patients to lead normal lives, treatment is considered burdensome and expensive

In the clinical trial, 132 otherwise healthy patients with hemophilia A received a one-time injection of valoccogene roxaparvovec and were monitored for more than a year

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