… 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 …
Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
… 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. …
… sur environ sept sites experts en Amérique du Nord et en Europe. Il s'agira d'une étude randomisée à double insu … et de la structure rétinienne au moyen de paramètres ophtalmiques tels que le champ visuel, l’acuité visuelle et la tomographie en cohérence optique. Les changements dans la qualité de vie des sujets de …
… six adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. … field stimulus testing (FST), ocular instability (OCI), optical coherence tomography (OCT), and pupillary light … global trial to be conducted at sites in North America and Europe with significant expertise in genetic retinal disease. …