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Secondary outcomes included safety and adverse event data, and change in MHFMS score for twelve versus six months of active treatment, body composition, quantitative SMN mRNA levels, maximum ulnar CMAP amplitudes, myometry and PFT measures

Gene Set Enrichment Analysis (GSEA): GSEA was performed using the clusterProfiler R package to identify enriched gene ontology (GO) terms associated with storage differences, sex differences, and QTL