Historical Control Analysis Demonstrates Greater Long‐Term Reduction in Plasma Globotriaosylceramide (Gb3) by Venglustat Compared With Placebo or Agalsidase Beta in Male Patients With Classic Fabry Disease
Venglustat showed significantly greater reductions in plasma GL-3 concentrations than placebo after 6 months and agalsidase beta after 24 and 36 months. These findings support the potential of long-term venglustat treatment to reduce GL-3 accumulation in patients with classic FD. Further studies are needed to confirm clinical benefit.
Authors
- Pronabesh DasMahapatra (ORCID: https://orcid.org/0000-0002-8242-0580)
- Patrick Deegan (ORCID: https://orcid.org/0000-0001-9427-9900)
- William R. Wilcox (ORCID: https://orcid.org/0000-0002-0325-0377)
- Vijay Modur (ORCID: https://orcid.org/0009-0001-3720-7553)
- Alberto Ortíz (ORCID: https://orcid.org/0000-0002-9805-9523)
- Dominique P. Germain (ORCID: https://orcid.org/0000-0002-8355-007X)
- Shiguang Liu (ORCID: https://orcid.org/0000-0003-0732-5230)
Institutions
- Emory University (US)
- Université de Versailles Saint-Quentin-en-Yvelines (FR)
- Université Paris-Saclay (FR)
- Cambridge University Hospitals NHS Foundation Trust (GB)
- Sanofi (United States) (US)
- Hospital Universitario Fundación Jiménez Díaz (ES)
- Universidad Autónoma de Madrid (ES)
Publication Details
- Journal
- Molecular Genetics & Genomic Medicine
- Published
- 2025-11-01
- DOI
- https://doi.org/10.1002/mgg3.70152
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Sanofi