Encouraging clinical data reported from Phase 1/2 trial of sepofarsen for LCA10
Initial clinical data from Phase 1/2 trial of QR-421a for Usher syndrome type 2 on track for Q1 2020
QR-1123 Investigational New Drug application active for autosomal dominant re
… of transformative RNA medicines for severe genetic rarediseases,today announced positive top-line results from … for the LCA10 community and the Inherited Retinal Disease community as a whole,” said Stephen R. Russell, MD, …
… RNA medicines for the treatment of severe genetic rarediseases,today reported its financial results for the … (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of …
… RNA medicines for the treatment of severe genetic rarediseases,today announced results for the first quarter … for Fuchs endothelial corneal dystrophy and Stargardt’s disease during the 2018 Annual Meeting of the Association for …
Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;Updated data from the Phase 1/2 InSight extension st
Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in vision; Phase 2/3 pivotal trial Illuminate initiated with data expected in H1 2021Phase 1/2 St