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Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients with Spinal Muscular Atrophy: The Phase 3b SMART Study
Hugh J. McMillan, Giovanni Baranello, Michelle A. Farrar, Craig M. Zaidman, Teresa Moreno, Liesbeth De Waele, Yuh Jyh Jong, Vincent Laugel, Susana Quijano-Roy, Eugenio Mercuri, Yin Hsiu Chien, Volker Straub, Basil T. Darras, Julia Seibert, Roberto Bernardo Escudero, Iulian Alecu, Frank Freischläger, Francesco Muntoni
Research output: Contribution to journal › Article › peer-review