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    1year
    Combined treatment with trametinib and riluzole improves motor function by enhancing autophagy in the SOD1-G93A mouse model of amyotrophic lateral sclerosis. (Neuroscience 2023)
    In motor neurons of trametinib+riluzole administrated SOD1-G93A mice, the level of cathepsin D and the co-localization of LC3 and LAMP1 increased, while abnormal p62 accumulation was significantly reduced, indicating improvement of autophagic flux. Altogether, these data suggest that low dose trametinib+riluzole combination therapy is pharmacologically superior to monotherapy in the ALS mouse model and could serve as a promising clinical combination for the improvement of current neuroprotective treatment strategies of ALS.
    Preclinical
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    LAMP1 (Lysosomal Associated Membrane Protein 1) • CTSD (Cathepsin D)
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    riluzole • trametinib repurposed (SNR1611)