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In addition, arimoclomol was shown to be safe and well tolerated in a double-blind, placebo-controlled trial performed with rapidly progressive SOD1-mutant ALS patients ( Ambroxol, a beta-glucocerebrosidase 2 (GBA2) inhibitor originally indicated as a generic expectorant and mucolytic drug to treat respiratory tract infectious disorders (Nobata et al., 2006), has been recently found to be a potential drug candidate for ALS treatment
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