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Article

Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials.

The British journal of ophthalmology - 1 Apr 2022

Daich Varela Malena, Cabral de Guimaraes Thales Antonio, Georgiou Michalis, Michaelides Michel

Abstract excerpt

Leber congenital amaurosis (LCA) is a severe congenital/early-onset retinal dystrophy. Given its monogenic nature and the immunological and anatomical privileges of the eye, LCA has been particularly targeted by cutting-edge research. In this review, we describe the current management of LCA, and highlight the clinical trials that are on-going and planned. RPE65-related LCA pivotal trials, which culminated in the...

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Identifiers

PMID: 33712480
DOI: 10.1136/bjophthalmol-2020-318483
PMCID: PMC8961750

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