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 HughesPatrick Deegan Pilar Giraldo Ozlem Goker-AlpanHeather LauElena LukinaShoshana Revel-VilkMaurizio ScarpaJaco BothaNoga GadirAri Zimrannull null

Published in: Journal of clinical medicine (2023)

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) [...].

Keyphrases

replacement therapy
machine learning
amino acid