Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)

作者：Derralynn Hughes, William Wilcox, Robert J. Hopkin, Jaya Ganesh, John Bernat, Özlem Göker-Alpan, Kathy Nicholls, Patrick Deegan, Madeleine V. Pahl, Chester B. Whitley, Michael Chen, Liching Cao, Katharina Schreeb · 发表于：Molecular Genetics and Metabolism · 年份：2025 · DOI：10.1016/j.ymgme.2024.108773 · 被引用次数：3 · 研究领域：Lysosomal Storage Disorders Research、Nuclear Receptors and Signaling