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In a study by Ain et al. (2025), eliglustat, an oral medication, was evaluated for its safety and effectiveness in treating children with type 1 Gaucher disease. The study involved 14 children who faced challenges with the standard intravenous treatment. They received eliglustat tailored to their genetic profiles for an average of 3.6 years. The results were promising, showing a significant decrease in disease activity markers, specifically glucosylsphingosine levels, with a statistical significance of p=0.005.
Additionally, the children experienced improvements in other disease indicators and reported better quality of life. Only minor side effects, like temporary gastroesophageal reflux, were observed in a few patients. The study concluded that eliglustat is a beneficial and safe alternative treatment for pediatric patients, mirroring the positive outcomes seen in adults.
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