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Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP-43 pathology.

Barış GençMukesh GautamÖge GözütokIna DervishiSantana SanchezGashaw M GoshuNuran KoçakEdward XieRichard B SilvermanP Hande Özdinler
Published in: Clinical and translational medicine (2021)
Mechanism-focused and cell-based drug discovery approaches not only addressed key cellular defects responsible for UMN loss, but also identified NU-9, the first compound to improve the health of diseased UMNs, neurons that degenerate in ALS, HSP, PLS, and ALS/FTLD patients.
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