Reply to Mistry et al. 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".

Derralynn A Hughes,Patrick Deegan,Pilar Giraldo,Özlem Göker-Alpan,Heather Lau,Elena Lukina,Shoshana Revel-Vilk,Maurizio Scarpa,Jaco Botha,Noga Gadir,Ari Zimran

Journal of clinical medicine（2023）

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摘要

Thank you for allowing us the opportunity to respond to the commentary from Mistry and colleagues (Comment: The two substrate reduction therapies for type 1 Gaucher disease are not equivalent) [...].

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gaucher disease,two substrate reduction therapies,gaucher outcome survey,substrate reduction therapies,enzyme replacement therapies

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