Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...
Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...
Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks ...
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