… “QR-1123 aims to block expression of the toxic mutated rhodopsin protein in the retina thereby targeting the … RHO ) gene. The trial will include single-dose escalation (open label) groups and multiple-dose escalation … function and retinal structure will be assessed through ophthalmic endpoints such as visual acuity, visual field and …
… 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 …
… 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 …
… (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 …
… conducted at about seven expert sites in North America and Europe. It will be a double-masked, randomized study … The first patient at each dose level will be dosed in an open-label manner. The objectives of the trial will include … pharmacokinetics and efficacy, as measured by stopping progression or improvement of visual function and …
… 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 to present QR-010 data at the European Cystic Fibrosis Society Conference and provides an … PQ-010-001, is expected to be completed in June 2017. Topline safety and exploratory efficacy data from the … QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a …