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Article

Establishing Clinical Trial Endpoints in Selecting Patients for RPGR Retinal Gene Therapy.

Translational vision science & technology - 3 Sept 2024

Christou Evita Evangelia, Josan Amandeep S, Cehajic-Kapetanovic Jasmina, MacLaren Robert E

Abstract excerpt

Purpose: Clinical trials for X-linked retinitis pigmentosa (RP) often assess retinal structure using optical coherence tomography (OCT) and function using microperimetry to evaluate initial eligibility and endpoints. Therefore, we seek to determine which parameters might be most sensitive in screening new patients for enrollment. Methods: Thirty-one patients (62 eyes) with confirmed retinitis pigmentosa GTPase...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 39287586
DOI: 10.1167/tvst.13.9.18
PMCID: PMC11412382

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