Microperimetry as an Outcome Measure Improves Patient Eligibility and Efficacy Detection in RPGR Gene Therapy Trials.

چکیده

To evaluate how primary endpoint selection influences patient eligibility, enrolment, and detection of treatment efficacy in RPGR gene therapy trials, comparing low luminance visual acuity (LLVA) with microperimetry-based outcome measures. Retrospective analysis of a phase 1/2 to 3 interventional…

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