… – LCA10 and sepofarsen ProQR Expert Perspectives Series on Leber congenital amaurosis 10 and sepofarsen with Aniz Girach … lun 20/07/2020 - 18:00 ProQR Expert Perspectives Series on Leber congenital amaurosis 10 and sepofarsen with Aniz Girach …
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.
… Announces Publication in Nature Medicine for Sepofarsen in Leber Congenital Amaurosis 10 LEIDEN, Netherlands & … with sepofarsen in a clinical trial for CEP290 mediated Leber congenital amaurosis 10 (LCA10). “We are excited to …
The EFF is a growing multiple stakeholder platform for the exchange and sharing of insights and innovations to drive the future of clinical development in inherited retinal diseases. Organized by ProQR
… 2/3 Illuminate trial of sepofarsen for CEP290 -mediated Leber Congenital Amaurosis 10 (LCA10) due to the p.Cys998X … are secondary endpoints in the Illuminate trial. About Leber Congenital Amaurosis 10 (LCA10) Leber congenital …
… trial results of intravitreal sepofarsen RNA therapy in Leber congenital amaurosis 10 (LCA10) Presenter: Stephen R. … testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis type 10 (LCA10) Presenter: Allen …
… call on July 20, 2020 at 5:00 pm EDT, which will focus on Leber congenital amaurosis 10 (LCA10) and the investigational … RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation …
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
QR-110 demonstrated rapid and sustained improvement in vision in the majority of subjects, as measured by visual acuity and mobility course
QR-110 was well-tolerated with no serious adverse events