Clinical efficacy analysis of disease-modifying therapies for spinal muscular atrophy with SMN1 gene compound heterozygous variants
DUAN Haolin, ZHANG Ciliu, XIONG Juan, PANG Nan, YIN Fei, PENG Jing
Journal of Clinical Pediatrics . 2025, (7): 543 -548 .  DOI: 10.12372/jcp.2025.24e1371