… Sepofarsen for CEP290 mediated Leber congenital amaurosis Sepofarsen is an investigational RNA therapy that aims to … mutation in the CEP290 gene. Sepofarsen (formerly named QR-110) is an investigational RNA therapy that aims to …
… amaurosis (LCA10). Learn more about the study and the sepofarsen program. Illuminate is a clinical study for Leber … pivotal trial is the second clinical study to investigate sepofarsen. This trial has completed enrollment. Please sign …
… aims to study whether the investigational RNA therapy sepofarsen is safe and well-tolerated in children younger … This trial is the fourth clinical study to investigate sepofarsen. If you are interested in joining this trial, …
… ProQR update and additional sepofarsen Illuminate trial analyses ProQR has announced the … unmet need. We are also sharing additional analyses of the sepofarsen *Illuminate* trial. ProQR has announced the …
… Expert Perspectives Series – LCA10 and sepofarsen ProQR Expert Perspectives Series on Leber congenital amaurosis 10 and sepofarsen with Aniz Girach and Dr. Ian M. MacDonald. This …
… ProQR Top-line results Phase 1/2 trial of sepofarsen in LCA10 patients ProQR presents Positive Top-Line Results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients. This event is virtually …
… Full field stimulus testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis … phase 1b/2 trial, 11 LCA10 patients received intravitreal sepofarsen . The goal was to evaluate patient response to …
… ARVO (2021) Russell SR et al., Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension … Russell from Iowa University on the Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension …