2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 13, 2021 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for
… 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 …
ProQR Therapeutics today announced virtual poster presentations at the Annual Meeting of the American Academy of Ophthalmology (AAO) held November 13-15, 2020.
Call on July 20 will include a snapshot from the ongoing InSight open-label extension study of sepofarsen, including preliminary second eye treatment data
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
LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced a virtual presentation of data fro
