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10.1002/14651858.CD001903.pub3 102 MasinoS

This review will focus on recent genetic discoveries in SMA, cellular mechanisms underlying motor neuron degeneration, understanding disease progression, and initiatives to address clinical trial readiness and the development of novel treatment strategies

[DOI] [PMC free article] [PubMed] [Google Scholar] 21.Kaczkan M., Czaja-Stolc S., Szczuko M., Drozd A., Rutkowski P., Dbska-lizie A., Magorzewicz S

qiang, Li, G., Zhi, X

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