STAAR, a phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Dose escalation phase results

Autor:	Robert J. Hopkin, Jaya Ganesh, Patrick Deegan, Ozlem Goker-Alpan, John Bernat, William R. Wilcox, Madeleine V. Pahl, Chester B. Whitley, Derralynn Hughes, Kathleen Nicholls, Liching Cao, Michael Chen, Lisa H. Shiue, Emma Bowden, Sravan Jaggumantri, Cristobal Passalacqua, Bernard Souberbielle, Bettina M. Cockroft
Rok vydání:	2023
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetics Molecular Biology Biochemistry
Zdroj:	Molecular Genetics and Metabolism. 138:107152
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2022.107152
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::2a2d91d5c71bb87b4993802f2bffe4a9 https://doi.org/10.1016/j.ymgme.2022.107152 Zobrazit plný text záznamu Full Text from ScienceDirect