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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.

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Published version
Peer-reviewed

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Authors

Mistry, Pramod K 
Kishnani, Priya S 
Balwani, Manisha 
Charrow, Joel M 
Hull, Judy 

Abstract

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

Description

Peer reviewed: True


Funder: Sanofi

Keywords

32 Biomedical and Clinical Sciences, Neurosciences, Orphan Drug, Rare Diseases, Neurodegenerative

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Publisher

MDPI AG