Harnessing transcriptomic signals for amyotrophic lateral sclerosis to identify novel drugs and enhance risk prediction.

Looking across two independent target expression datasets, the association between ALS PTS and case-control status was highly significant, explaining up to 5.1 % of ALS risk on the liability scale.

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Of the drugs identified by GCSC, TWAS-GSEA found nominally significant evidence across two expression panels that Oxymetholone induces an expression signature that may increase risk of ALS.

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