Isaralgagene civaparvovec (ST-920) shows positive mean annualized eGFR slope in adults with Fabry disease: Topline results from the registrational phase 1/2 STAAR gene therapy study and long-term follow-up study

Molecular Genetics and Metabolism 2026

View at publisher
Authors: John A. Bernat, Robert James Hopkin, William R. Wilcox, Derralynn A. Hughes, Jaya Ganesh, Özlem Göker-Alpan, Kathy Nicholls, Patrick B. Deegan, Liching Cao, Michael Chen, Katharina H. Schreeb
DOI: 10.1016/j.ymgme.2025.109334
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Glycogen Storage Diseases and Myoclonus

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109334–109334
Publication date
1 February 2026
Language
EN