共 50 条
- [6] An evaluation of onasemnogene abeparvovec for spinal muscular atrophy (SMN1) EXPERT OPINION ON ORPHAN DRUGS, 2021, 9 (7-10): : 199 - 204
- [8] Spinal muscular atrophy carriers with two SMN1 copies BRAIN & DEVELOPMENT, 2017, 39 (10): : 851 - 860