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
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