Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy.

Mol Ther Methods Clin Dev · 2025 · PMC12127541 · PMID 40458203

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did not reach statistical significanceno p-value reported

Although the reduction in transgene expression in the liver did not reach statistical significance owing to the low n in each group ( n = 4 animals per group), a downward trend was observed in both mRNA and protein levels.

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