학술논문
Onasemnogene Abeparvovec Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with 3 Copies of SMN2
Document Type
Journal
Author
Strauss, Kevin; Muntoni, Francesco; Farrar, Michelle; Saito, Kayoko; Mendell, Jerry; Servais, Laurent; McMillan, Hugh; Swoboda, Kathryn; Kwon, Jennifer; Zaidman, Craig; Chiriboga, Claudia; Iannaccone, Susan; Krueger, Jena; Parsons, Julie; Shieh, Perry; Kavanagh, Sarah; Chand, Deepa; Tauscher-Wisniewski, Sitra; Macek, Thomas
Source
Subject
Language
English
ISSN
1526632X