Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, ...
Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...
Variations in a DNA building block of genes tied to processes like inflammation associated in a study with disease type, ...
Scientists sought to understand the frequency of severe respiratory tract infections in SMA children, notably those needing ...
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