The clinical trial named Brighten evaluates the safety and tolerability of investigational RNA therapy sepofarsen in children with Leber congenital amaurosis.
A blog by Martin Hills from Retina UK living with Retinitis Pigmentosa on why research participation and genetic testing are essential in for drug development and how to get involved.
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 seve
ProQR Therapeutics today announced positive results from a planned analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations.
In a Phase 1/2 clinical trial investigational RNA therapy QR-421a showed a benefit in vision. ProQR plans to start final phase trials for USH2A mediated retinitis pigmentosa.
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.
The ProQR family is growing fast and we spoke to the Chief Medical Officer Aniz Girach who joined ProQR in 2019. We asked him about his impression of the company after he joined, as well as the new developments he’s been working on.
During a ProQR Expert Perspective call Dr. Girach (CMO at ProQR) discusses findings from an open label extension study of sepofarsen for LCA10 with Retinal Disease expert Dr. MacDonald (Prof Emer University of Alberta).
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra
