… 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 …
… ProQR has granted an exclusive license to Amylon to develop therapeutics for beta amyloid related disorders and … to future milestones and royalties from the products developed. Amylon will initially focus on developing a RNA-based therapy for a rare genetic disease …
… in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, … 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients … expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and …
… are no therapies commercially available The Phase 1/2 open-label trial will assess the safety, tolerability, … 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients … expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and …
… PQ-110-001 Phase 1/2 Trial PQ-110-001 is a first-in-human open-label trial that enrolled 5 children (age 6 - 17 years) … and 6 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 (BCVA), mobility …