… retinal disease, into the clinic and continuing the development of our portfolio of transformative RNA medicines … ProQR. “QR-421a has shown promising activity in both the optic cup and zebra fish models and we are excited about the … gene and will be conducted at expert sites in the US and Europe. It will be a double-masked, randomized study …
… 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 …
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.
… 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 …