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 May 4;12(9):3269.
doi: 10.3390/jcm12093269.
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.