The era of cryptic exons: implications for ALS-FTD.

Mol Neurodegener · 2023 · PMC10018954 · PMID 36922834

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failed to reach significanceno p-value reported

The former showed very encouraging results, but failed to reach significance in achieving primary clinical endpoints in a phase 3 trial, and the latter phase 1 trial showed no difference from placebo for clinical endpoints and in fact trended towards greater decline at the higher dose.

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