Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives.
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
ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
H.C. Wainwright Liver Disease Virtual Conference – October 21-22, 2025
Presentation available on demand beginning October 21 at 7:00 am ET and virtual 1x1s on October 22
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.