Article

Identifying trial-relevant concepts of interest in HSP: insights from an international patient-voice study in over 600 individuals

2026-04-10

Abstract excerpt

<h4>Background</h4> As therapeutic options emerge for hereditary spastic paraplegias (HSP), clinical trials require outcome measures that reflect disease aspects most important to patients. Patient priorities in HSP remain poorly defined. This study aimed to develop a regulatory-compliant framework of patient-prioritised health domains to evaluate treatment response in clinical trials. <h4>Methods</h4> Patient-r...

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Literature Corpus work: 524221c5-f01c-563d-8504-fb752fbeb0c4
DOI: 10.64898/2026.04.09.26350392

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