Challenges in Gaucher disease: Perspectives from an expert panel
Grabowski G, Kishnani P, Alcalay R, Prakalapakorn S, Rosenbloom B, Tuason D, Weinreb N. Challenges in Gaucher disease: Perspectives from an expert panel. Molecular Genetics And Metabolism 2025, 145: 109074. PMID: 40112481, DOI: 10.1016/j.ymgme.2025.109074.Peer-Reviewed Original ResearchGD type 1Enzyme replacement therapyNeuronopathic diseaseGD type 2Type 1Central nervous systemMagnetic resonance imagingGaucher diseaseDixon quantitative chemical shift imagingFirst-line treatment of adultsEnzyme replacement therapy productsDual-energy X-ray absorptiometry scanPrompt initiation of treatmentEffects of enzyme replacement therapyX-ray absorptiometry scanQuantitative chemical shift imagingSymptoms of central nervous systemGD type 1 patientsGD type 3Non-Hodgkin's lymphomaPeak bone massFirst-line treatmentType 2Nervous systemInitiation of treatment
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