Paganoni S, Macklin EA, Hendrix S, et al. - This multicenter, randomized, double-blind trial was undertaken to test the efficacy as well as the safety of a combination of sodium phenylbutyrate and taurursodiol in patients with amyotrophic lateral sclerosis (ALS). Patients with definite ALS who had had an onset of symptoms within the previous 18 months were enrolled in this study. Randomization of patients was done in a 2:1 ratio to receive sodium phenylbutyrate–taurursodiol (3 g of sodium phenylbutyrate and 1 g of taurursodiol, given once a day for 3 weeks and then twice a day) or placebo. As per findings, slower functional decline was observed in relation to treatment with sodium phenylbutyrate–taurursodiol vs placebo as determined by the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised score over a span of 24 weeks. Between the two groups, no significant difference was evident in secondary outcomes, which were the rates of reduction in isometric muscle strength, plasma phosphorylated axonal neurofilament H subunit concentrations, and the slow vital capacity; the time to death, tracheostomy, or permanent ventilation; and the time to death, tracheostomy, permanent ventilation, or hospitalization.