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The results showed that all 15 patients were alive and event-free at 20 months of age, compared to a survival rate of 8 in a historical cohort. In the high-dose group, a rapid increase in the CHOP INTEND scale score was observed after gene delivery, with an increase of 9. 8 points at the first month and 15. 4 points at 3 months, compared to with a decrease in this score in the historical group. Of the 12 individuals who received the greatest amount of vector, 11 achieved physical improvements such as sitting unassisted, 9 rolled over, 11 fed orally and were able to speak, and 2 walked independently. Finally, elevated serum aminotransferase levels were evident in 4 people, which were attenuated by prednisolone. Depuis 2006, ce centre hospitalier a admis plus de 4 224 patients et menГ plus de 9 000 opГrations chirurgicales. Ces cas sont repГrГs par un rГseau dвagents de liaison mГdicale dans les pays dвorigine des patients.
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