… 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 …
… (over eight years of age) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene and a … full field stimulus testing, ocular instability and optical coherence tomography. Changes in quality of life in … to be conducted at sites in North America and selected European countries with significant expertise in genetic …
… ProQR spins out all Dystrophic Epidermolysis Bullosa activities into newly formed … EBRP to apply our experience in DEB drug development to develop transformational therapies for DEB patients,” said … a loss of the anchoring fibrils resulting in fragile skin. People with DEB live with constant pain and have a high risk …