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ABSTRACT: Background
X-linked retinitis pigmentosa (XLRP) is a rare, inherited retinal disease characterized by impairment in visual field and visual acuity with continuous progression leading to blindness. Gene therapies for XLRP are under investigation, and health state utilities are needed for use in cost-utility analyses examining the value of these treatments.Objective
This study aimed to estimate utilities associated with XLRP severity.Methods
Eleven health state vignettes depicting combinations of impairment in visual field and visual acuity associated with XLRP were developed based on literature review and input from clinicians, patients, and a caregiver. Vignettes included text describing visual acuity impairment, visual field impairment, night blindness, impact on q
SUBMITTER: Matza LS
PROVIDER: S-EPMC12431885 | biostudies-literature | 2025 Sep
REPOSITORIES: biostudies-literature