Therapeutic frontiers in ALS: iPSC-based drug discovery, cell therapy, and gene therapy-Advances through 2026.

Regen Ther · 2026 · PMC13292465 · PMID 42359165

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an encouraging trendno p-value reported

Interim results announced by the company in February 2026 reported confirmed target engagement with statistically significant STMN2 increases, correction of STMN2 mis-splicing, statistically significant pNfH reduction, and an encouraging trend of slowed ALSFRS-R decline.

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