… pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene. P23H is the most prevalent mutation … gene. The trial will include a single-dose escalation (open label) arm and a multiple-dose (double-masked) arm in … of visual function and retinal structure through ophthalmic endpoints such as visual acuity (BCVA), visual …
… Presentations”. About PQ-110-001 trial PQ-110-001 is an open-label trial that has been designed to enroll … six adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients … of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field …