… 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 …
Presentation at the 18th International Symposium on Retinal Degeneration (RD2018) at 8:30 am GMT on Sept. 5th Investor conference call to be held at 8:15 am ET on Sept. 5th
… (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 …
… trial of QR-010 in CF patients with the F508del mutation. Top-line trial data are expected to be issued in a press … QR-010 is designed to be self-administered via an optimized eFlow ® Nebulizer (PARI Pharma GmbH). eFlow ® is a … orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 …
… 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 …