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

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:2023-05-04 Issue:9 Volume:12 Page:3269
ISSN:2077-0383
Container-title:Journal of Clinical Medicine
language:en
Short-container-title:JCM

Author:

Mistry Pramod K.¹,Kishnani Priya S.²,Balwani Manisha³,Charrow Joel M.⁴,Hull Judy⁵,Weinreb Neal J.⁶^ORCID,Cox Timothy M.⁷^ORCID

Affiliation:

1. Department of Medicine, Pediatrics, and Cellular & Molecular Physiology, Yale University School of Medicine, 20 York Street, New Haven, CT 06510, USA

2. Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA

3. Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA

4. Division of Genetics, Genomics, and Metabolism, Northwestern University Feinberg School of Medicine, Ann & Robert H Lurie Children’s Hospital of Chicago, Chicago, IL 60611, USA

5. Gaucher Disease, US Medical Affairs, Sanofi, Cambridge, MA 02141, USA

6. Department of Human Genetics, University of Miami Miller School of Medicine, Miami, FL 33433, USA

7. Lysosomal Disorders Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge CB2 0QQ, UK

Abstract

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

Funder

Sanofi

Publisher

MDPI AG

Subject

General Medicine

Link

https://www.mdpi.com/2077-0383/12/9/3269/pdf

Reference19 articles.

1. 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.

2. A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration;Peterschmitt;Blood Cells Mol. Dis.,2018

3. (2018). CERDELGA™ (Eliglustat) [Package Insert], Genzyme Ireland, Ltd.. Available online: http://www.cerdelga.com/pdf/cerdelga_prescribing_information.pdf.

4. Summary of Product Characteristics, Genzyme Therapeutics. Available online: https://www.medicines.org.uk/emc/product/2615/smpc2018.

5. (2017). Zavesca (Miglustat) US Prescribing Information, Actelion Pharmaceuticals US, Inc. Available online: https://www.zavesca.com/pdf/ZAVESCA-Full-Prescribing-Information.pdf.

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3. 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”;Journal of Clinical Medicine;2023-06-13