Researchers working in four different countries have published the final data from three phases of clinical trials on gene therapy for Wiskott-Aldrich syndrome, as well as the results of long-term monitoring of study participants.
According to the presented data, patients who underwent treatment experienced a significant reduction in the frequency of serious bleeding and infectious diseases. Furthermore, five years after starting therapy, all subjects, with the exception of one person, remained alive.
The full report on the conducted trials was officially published in the scientific journal The New England Journal of Medicine.