关键词: Gene therapy Nusinersen Onasemnogene abeparvovec SMN1 SMN2 Spinal muscular atrophy Zolgensma
Mesh : Biological Products / therapeutic use Consensus Genetic Therapy / methods Humans Infant Muscular Atrophy, Spinal / genetics therapy Patient Selection Recombinant Fusion Proteins / therapeutic use
来 源:
DOI:10.1016/j.ejpn.2020.07.001
PDF(Sci-hub)
PDF(Pubmed)