AAO 2020 poster by Allen Ho et al. Recognized with best poster award. Data show that FST seems a more sensitive method in patients with severely impaired vision.
… in the second treated eye in a Ph1b/2 extension trial in Leber Congenital Amaurosis type 10 (LCA10) ARVO 2021 … treated eye in a Ph1b/2 extension trial in CEP290 mediated Leber Congenital Amaurosis type 10. Author(s) Stephen R. …
AAO 2020 poster by Stephen Russel et al. Recognized with best poster award. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
… 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 …
… 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
