Work

Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale

Year: 2025

Type: article

Abstract: The placebo response in clinical trials in ataxias complicates outcome interpretation and potentially obscures genuine treatment effects. We analyzed placebo group data from past trials in Friedreich Ataxia and observed notable responses in appendicular items, in contrast to minimal changes in axial function, as measured by respective subscores of the modified Friedreich Ataxia Rating Scale (mFARS). The effect increased with the number of consecutive tests, shorter testing intervals, and older group ages. This has implications for trial design and endpoint selection, thus strengthening the utility of the Upright Stability Score (USS), a sub-score of mFARS, as an independent measure.

Source: Annals of Clinical and Translational Neurology

DOI: 10.1002/acn3.70239

Authors: Christian Rummey, Jennifer M. Farmer, David R. Lynch

Institutions: Friedreich's Ataxia Research Alliance, Children's Hospital of Philadelphia

Language: English

Topic: Pain Management and Placebo Effect

Subfield: Cognitive Neuroscience

Field: Neuroscience

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $4,100