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Gaucher disease (GD), a rare genetic disorder inherited via the autosomal recessive mode, is the most prevalent lysosomal storage disorder (1). Mutations in the glucocerebrosidase (GBA) gene lead to GD manifestations (1). Nearly 300 GBA mutations have been identified, including missense, nonsense, splice junction, deletion, insertion, gene fusion,
<div><strong>Transforming Gaucher Disease Care: Venglustat Clinical Data Highlights</strong></div>

Transforming Gaucher Disease Care: Venglustat Clinical Data Highlights

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