Analysis of sodium phenylbutyrate and taurursodiol survival effect in ALS using external controls.
Sabrina PaganoniMelanie QuintanaAlexander V ShermanMatteo VestrucciYuehui WuJamie TimmonsMerit E Cudkowicznull nullPublished in: Annals of clinical and translational neurology (2023)
This analysis suggests potentially greater survival benefit with PB and TURSO in ALS without placebo-to-active crossover than seen on ITT analysis in CENTAUR. Analyses using well-matched external controls may provide additional context for evaluating survival effects in future ALS trials.