학술논문
STAAR, a phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Dose escalation phase results.
Document Type
Article
Author
Hopkin, Robert J.; Ganesh, Jaya; Deegan, Patrick; Goker-Alpan, Ozlem; Bernat, John; Wilcox, William R.; Pahl, Madeleine V.; Whitley, Chester B.; Hughes, Derralynn; Nicholls, Kathleen; Cao, Liching; Chen, Michael; Shiue, Lisa H.; Bowden, Emma; Jaggumantri, Sravan; Passalacqua, Cristobal; Souberbielle, Bernard; Cockroft, Bettina M.
Source
Subject
*ANGIOKERATOMA corporis diffusum
*GENE therapy
*ADULTS
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Language
ISSN
1096-7192