Article

Development of patient-reported outcome for spinal and bulbar muscular atrophy

2026-07-06

Abstract excerpt

<h4>Objectives</h4> To develop and validate a disease-specific patient-reported outcome (PRO) measure for spinal and bulbar muscular atrophy (SBMA). <h4>Methods</h4> A three-stage sequential design was adopted. Items were generated through qualitative interviews with patients with SBMA and expert review, refined using quantitative analyses, and evaluated for reliability and validity in independent cohorts from J...

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Literature Corpus work: 5f6db10d-fc04-5b78-a37a-4436e7f26f0b
DOI: 10.64898/2026.07.02.26356783

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