… are no therapies commercially available or in clinical development for the vision loss associated with this disease. … preclinical data in both patient fibroblasts and the optic cup model for mRNA restoration, which was presented at … of the Association for Research in Vision and Ophthalmology (ARVO) in May 2017. A lead candidate has been …
… lives through transformative RNA therapies based on its proprietary Axiomer ™ RNA editing technology platform, today … addition, ProQR has granted the underwriters a 30-day option to purchase up to 2,700,000 additional ordinary shares … prevalent diseases with unmet need. Based on its unique proprietary RNA repair platform technologies ProQR is growing …
… Partnership focused on utilizing Axiomer ™ to develop editing oligonucleotides targeting an underlying … Collaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases … lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today …
The trial, called WINGS, will evaluate the safety and efficacy of QR-313 in subjects with recessive dystrophic epidermolysis bullosa due to mutations in exon 73 of the COL7A1 gene.
… cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced its support … Since its inception in 2008, led by EURORDIS-Rare Diseases Europe and its Council of National Alliances, thousands of … common. LCA 10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To …
… ProQR to Present its Ophthalmology Pipeline at ARVO, Highlighting Programs in … data for three programs targeting forms of genetic retinopathy resulting in blindness. For ProQR’s lead ophthalmic program, QR-110 for Leber’s congenital amaurosis …
Le QR-110 a permis une amélioration rapide et soutenue de la vision chez la majorité des sujets, tant en termes d’acuité visuelle que de mobilité Le QR-110 a été bien toléré sans événements indésirables graves