LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 04, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S.
… pigmentosa (adRP) due to the P23H mutation in the rhodopsin ( RHO ) gene. Fast Track designation is granted … ) 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 …
… 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 …
… 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 …
… L’Agence européenne des médicaments accorde l’accès PRIME au … et seulement 20 % (une sur cinq) des demandes en ophtalmologie l'avaient été. « La décision de l’EMA … prometteuses de son programme principal en ACL10. À propos du Sepofarsen Le Sepofarsen est le premier …
… ProQR Initiates Phase 1/2 Clinical Trial of QR-313 for Dystrophic Epidermolysis Bullosa The trial, called WINGS, … with recessive dystrophic epidermolysis bullosa (RDEB) is open for enrollment. “The initiation of our first human … clinical trial for QR-313 is an exciting next step in the development of this novel investigational therapy for DEB. …
… 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 …