Kim A. Staats

The paper identifies a multi-protein complex in PBMCs as a target for ALS therapeutic assembly modulators, showing altered composition in ALS patients and responsiveness to treatment.

This paper introduces a new drug-like small molecule series targeting PDI in a multi-protein complex for ALS treatment, showing activity in cellular and animal models.

This study shows that inhibiting PIKFYVE kinase rescues both gain- and loss-of-function C9ORF72 disease mechanisms in mouse models of ALS/FTD.