… two abstracts on programs for Fuchs endothelial corneal dystrophy and Stargardt’s disease at the ARVO annual meeting. … patients have been enrolled in PQ-110-001, a Phase 1/2 open-label trial assessing the safety, tolerability, … treatment to LCA 10 patients that have no other treatment options,” said Daniel A. de Boer, Chief Executive Officer of …
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 …