Aniz Girach, MD, Chief Medical Officer, and Paul Yang, MD, PhD, Casey Eye Institute will discuss which vision measures are most informative in Usher syndrome, the role of baseline and disease progression, and objectives of the Stellar trial.
The EFF Spring Virtual Summit hosted by ProQR on April 15 is a virtual meeting featuring the latest clinical data for USH2A mediated Usher syndrome and retinitis pigmentosa and a live panel discussion.
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
Company management will present and discuss results from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP).
On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.
ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).