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Analysis of sodium phenylbutyrate and taurursodiol survival effect in ALS using external controls.

Sabrina PaganoniMelanie QuintanaAlexander V ShermanMatteo VestrucciYuehui WuJamie TimmonsMerit E Cudkowicznull null
Published 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.
Keyphrases
  • amyotrophic lateral sclerosis
  • free survival
  • randomized controlled trial
  • heavy metals
  • open label
  • current status
  • placebo controlled
  • phase iii