Eliglustat substrate reduction therapy in children with Gaucher disease type 1

Eliglustat substrate reduction therapy in children with Gaucher disease type 1

ImportanceGaucher disease (GD) is a rare lysosomal storage disorder with limited treatment options for pediatric patients. Oral substrate reduction therapy (SRT) with eliglustat offers a potential alternative, particularly for those with barriers to enzyme replacement therapy (ERT).ObjectiveEvaluate...

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Bibliographic Details
Main Authors: Noor Ul Ain, Armaan Saith, Audrey Ruan, Ruhua Yang, Aaron Burton, Pramod K. Mistry
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-02-01
Series:Frontiers in Pediatrics
Subjects:
Gaucher disease (GD)
precision medicine
substrate reduction therapy (SRT)
eliglustat
lysosomal storage disease (LSD)
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2025.1543136/full
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