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Preclinical assessment of GNE<sup>wt</sup>/bi-shRNA-GNE<sup>M743T</sup> lipoplex product development for GNE myopathy.

Future Sci OA · 2026 · PMC12962615 · PMID 41770218

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showed a trendno p-value reported
This study did not demonstrate superior muscle strength compared to the placebo group, however biological activation through significant re-sialylation of cells showed a trend toward slowed muscle fat degeneration on MRI.

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