What are the Potential Benefits of Eliglustat for Pediatric Patients?
For pediatric patients with type 1 Gaucher disease, eliglustat offers a potential alternative to enzyme replacement therapy (ERT), which is the standard treatment. Eliglustat's oral administration can be more convenient and less invasive compared to the intravenous infusions required by ERT. Additionally, eliglustat may help in reducing the frequency and severity of symptoms by inhibiting the accumulation of glucocerebroside.