… 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 …
… 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 …
… difference (NPD). This proof-of-concept study is an open label 28-day study conducted in 5 specialized centers in the US and Europe. The study plans to enroll 16 CF patients, 8 homozygous (carrying two copies) for the ΔF508 mutation and 8 compound heterozygous …
… 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 …
… cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced details … Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, will give an oral … QR-010 is designed to be self-administered via an optimized eFlow ® Nebulizer (PARI Pharma GmbH). eFlow ® is a …