Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy.

Mol Ther · 2019 · PMC6453518 · PMID 30879951

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did not reach statistical significanceno p-value reported

The differences for biomarker clearance in the kidney between the two modalities, ERT and MRT, did not reach statistical significance, representing equivalent reduction for each, even though MRT was dosed only on a monthly regimen.

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