Exenatide Once Weekly in the Treatment of Patients with Multiple System Atrophy.

ABSTRACT:

Objective

Exenatide, a glucagon-like peptide-1 (GLP-1) receptor agonist, has neuroprotective effects in preclinical models of multiple system atrophy (MSA). We investigated these effects in a proof-of-concept clinical trial.

Methods

In this single-center, randomized, open label trial, participants with MSA were randomly assigned (1:1) to receive subcutaneous injections of exenatide 2 mg weekly for 48 weeks, or as controls, followed by a 48-week washout period. The primary outcome was the Unified Multiple System Atrophy Rating Scale (UMSARS) parts I + II combined score at 48 weeks. Objective secondary outcome measures included the numbers of participants losing ambulation; scoring ≥ 3 on UMSARS part I items for falls, speech, swallowing, as well as timed walking and measures

SUBMITTER: Vijiaratnam N 

PROVIDER: S-EPMC12577669 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature