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”-Reference-Cited by-同舟云学术

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”

Published:2023-06-13 Issue:12 Volume:12 Page:4017
ISSN:2077-0383
Container-title:Journal of Clinical Medicine
language:en
Short-container-title:JCM

Author:

Hughes Derralynn A.¹,Deegan Patrick²^ORCID,Giraldo Pilar³⁴^ORCID,Göker-Alpan Özlem⁵^ORCID,Lau Heather⁶,Lukina Elena⁷,Revel-Vilk Shoshana⁸⁹^ORCID,Scarpa Maurizio¹⁰,Botha Jaco¹¹,Gadir Noga¹¹,Zimran Ari⁸⁹^ORCID

Affiliation:

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) [...]

Funder

The Gaucher Outcome Survey (GOS) is sponsored by Takeda Pharmaceutical Company Limited.

Publisher

MDPI AG

Subject

General Medicine

Link

https://www.mdpi.com/2077-0383/12/12/4017/pdf

Reference2 articles.

1. Mistry, P.K., Kishnani, P.S., Balwani, M., Charrow, J.M., Hull, J., Weinreb, N.J., and Cox, T.M. (2023). 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., 12.

2. Hughes, D.A., Deegan, P., Giraldo, P., Göker-Alpan, Ö., Lau, H., Lukina, E., Revel-Vilk, S., Scarpa, M., Botha, J., and Gadir, N. (2022). Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med., 11.