Isaralgagene civaparvovec (ST-920) gene therapy for adults with Fabry disease: Pharmacology and immunogenicity outcomes from the phase 1/2 STAAR study and ongoing long-term follow-up

Molecular Genetics and Metabolism 2026

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Authors: Yanmei Lu, Liching Cao, Yonghua Pan, Jing Hu, Michael Chen, Kathy E. Meyer

DOI: 10.1016/j.ymgme.2025.109530

Cited by: 0

Fields of Study & Subjects

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