Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence
Des améliorations rapides, significatives et durables de la vision à douze mois ont été rapportéesAmélioration concordante des mesures des résultats secondaires clésLa dose d'enregistrement cible du sepofarsen a été bien tolérée avec un profil risque/bénéfice fa
… board, where his expertise will help guide and inform our development of therapies for rare genetic diseases based on … ® technology." Dr. Yu is a professor of biochemistry and biophysics at the University of Rochester Medical Center. His … rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. Based …
… NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR), a biopharmaceutical company dedicated to changing lives through … In addition, ProQR has granted the underwriters a 30-day option to purchase up to 862,500 additional ordinary shares … for the offering. Kempen & Co is acting as the Company’s European financial advisor in relation to this offering. A …
… NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a biopharmaceutical company dedicated to changing lives through … offering, ProQR has granted the underwriters a 30-day option to purchase up to 745,471 additional ordinary shares … as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique …
… lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today … The 2023 edition of the RNA Editing Summit will focus on optimizing RNA editing technology to safely deliver novel …
… addition, ProQR has granted the underwriters a 30-day option to purchase up to 2,076,923 additional ordinary shares … the offering has been filed with the SEC. When available, copies of the final prospectus supplement and the … syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing …
… 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 …