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; GOS Steering Committee

doi:10.3390/jcm12124017

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"

J Clin Med. 2023 Jun 13;12(12):4017. doi: 10.3390/jcm12124017.

Authors

Derralynn A Hughes¹, Patrick Deegan², Pilar Giraldo^{3

4}, Özlem Göker-Alpan⁵, Heather Lau⁶, Elena Lukina⁷, Shoshana Revel-Vilk^{8

9}, Maurizio Scarpa¹⁰, Jaco Botha¹¹, Noga Gadir¹¹, Ari Zimran^{8

9}; GOS Steering Committee

Affiliations

¹ LSD Unit, Royal Free London NHS Foundation Trust, University College London, London NW3 2QG, UK.
² Addenbrookes Hospital, Cambridge CB2 0QQ, UK.
³ CIBER de Enfermedades Raras, IIS Aragon, 50009 Zaragoza, Spain.
⁴ Translational Research Unit, IIS Aragon, 50009 Zaragoza, Spain.
⁵ Lysosomal Disorders Unit and Center for Clinical Trials, O&O Alpan LLC, Fairfax, VA 22030, USA.
⁶ Department of Neurology, New York University School of Medicine, New York, NY 10016, USA.
⁷ Department of Orphan Diseases, National Research Center for Hematology, 125167 Moscow, Russia.
⁸ Gaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
⁹ The Faculty of Medicine, Hebrew University, Jerusalem 9112102, Israel.
¹⁰ Centre for Rare Diseases, Academic Medical Centre Hospital of Udine, 33100 Udine, Italy.
¹¹ Takeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.

Abstract

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) [...].

Grants and funding

R and D contracts with each site/The Gaucher Outcome Survey (GOS) is sponsored by Takeda Pharmaceutical Company Limited.