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  2. ProQR Receives Fast Track Designation from FDA for QR-010 for Cystic Fibrosis

    Press release

    … (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 …

  5. ProQR to Present Interim Phase 1/2 Results for QR-110 in Leber’s Congenital Amaurosis 10 at the Symposium for Retinal Degeneration on Sept. 5th

    Press release

    … 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 …

  6. ProQR Doses First Patient in Phase 2/3 ILLUMINATE Trial of Sepofarsen for LCA10

    Press release

    … (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 …