NewsJOURNAL OF CLINICAL MEDICINESaturday, March 28, 2026 · March 28, 2026
Real-World Effectiveness and Safety of Eliglustat in Adult Patients with Gaucher Disease Type 1: A Multicenter Retrospective Study in China.
WHY IT MATTERS
Recent peer-reviewed research on Gaucher disease that may be relevant for patients and caregivers.
Background/Objectives: Eliglustat is an oral therapy for Gaucher disease type 1 (GD1) that may reduce infusion-related logistical burden, particularly in resource-constrained settings. Post-approval evidence from routine clinical practice in China remains limited. This study evaluated its real-world...
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