… Therapeutics, will present on June 20, 2024 at 2:00pm ET: “Developing Axiomer RNA Editing Technology Towards Clinical Development” This presentation will provide an overview of ProQR’s proprietary ADAR-mediated Axiomer RNA editing technology …
… Board. Dr. Dryja will play a key strategic role in the development and advancement of ProQR’s growing ophthalmology pipeline. LEIDEN, The Netherlands, Nov. 09, …
… (FDA) for its molecule QR-010 that is in clinical development for the treatment of patients with CF due to the … CFTR function, the nasal potential difference (NPD). This open label 28-day study enrolls 16 CF patients, 8 homozygous (carrying two copies) for the ΔF508 mutation and 8 compound heterozygous …
… pigmentosa (adRP) due to the P23H mutation in the rhodopsin ( RHO ) gene. ODD provides a special status for … ) 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 …