New England Journal of Medicine - Trial of Sodium Phenylbutyrate - Taurursodiol for ALS

Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis


Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy and safety of a combination of the two compounds in persons with ALS are not known.


Sodium phenylbutyrate–taurursodiol resulted in slower functional decline than placebo as measured by the ALS Functional Rating Scale–Revised score over a period of 24 weeks. Secondary outcomes were not significantly different between the two groups. Longer and larger trials are necessary to evaluate the efficacy and safety of sodium phenylbutyrate–taurursodiol in persons with ALS.