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The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158

Pramod K. Mistry, Priya S. Kishnani, Manisha Balwani, Joel M. Charrow, Judy Hull, Neal J. Weinreb, Timothy M. Cox

2023Journal of Clinical Medicine12 citationsDOIOpen Access PDF

Abstract

In their paper, Hughes et al. [...].

Topics & Concepts

MedicineEnzyme replacement therapySubstrate reduction therapyDiseaseReduction (mathematics)Gaucher's diseaseSubstrate (aquarium)Internal medicineMathematicsGeometryGeologyOceanographyLysosomal Storage Disorders ResearchCellular transport and secretionTrypanosoma species research and implications
The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158 | Litcius