Biological effects of sodium phenylbutyrate and taurursodiol in Alzheimer's disease.

ABSTRACT:

Introduction

Sodium phenylbutyrate and taurursodiol (PB and TURSO) is hypothesized to mitigate endoplasmic reticulum stress and mitochondrial dysfunction, two of many mechanisms implicated in Alzheimer's disease (AD) pathophysiology.

Methods

The first-in-indication phase 2a PEGASUS trial was designed to gain insight into PB and TURSO effects on mechanistic targets of engagement and disease biology in AD. The primary clinical efficacy outcome was a global statistical test combining three endpoints relevant to disease trajectory (cognition [Mild/Moderate Alzheimer's Disease Composite Score], function [Functional Activities Questionnaire], and total hippocampal volume on magnetic resonance imaging). Secondary clinical outcomes included various cognitive, functional, and neurops

SUBMITTER: Arnold SE 

PROVIDER: S-EPMC11310855 | biostudies-literature | 2024 Jul-Sep

REPOSITORIES: biostudies-literature