Doenças raras na infância: avanços no tratamento da Doença de Fabry com a terapia gênica ST-920

Referências

1. 1. Hopkin, R. J., Ganesh, J., Bernat, J., Goker-Alpan, O., Nicholls, K., Pahl, M. V., … & Wilcox, W. R. (2024, February). Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR). In Molecular Genetics and Metabolism (Vol. 141, No. 2). 525 B ST, STE 1900, SAN DIEGO, CA 92101-4495 USA: ACADEMIC PRESS INC ELSEVIER SCIENCE. 

2. Ganesh, J., Deegan, P., Goker-Alpan, O., Hopkin, R. J., Bernat, J. A., Wilcox, W., … & Cockroft, B. M. (2022). Preliminary results of STAAR, a Phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease and long-term follow-up. In Annu Symp Soc Study Inborn Errors Metab (SSIEM).