ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
Access based on positive interim analysis of clinical data as well as preclinical data to date PRIME designation provides a pathway for frequent and early interactions with the EMA aimed at supporting accelerated evaluation and approval
Divestment of sepofarsen and ultevursen completed – Théa to continue development of sepofarsen and ultevursen for patients with LCA10 and Usher syndrome
… Receives Rare Pediatric Disease Designation from FDA for Sepofarsen for the Treatment of LCA10 LEIDEN, Netherlands & … from the U.S. Food and Drug Administration (FDA) for sepofarsen for the treatment of Leber’s congenital amaurosis …
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