At ProQR science is literally at the heart of what we do. Our passion lies in discovering the latest RNA technologies and translating them to life-changing treatments.
… two dosing cohorts and is intended to inform next steps in development. We also plan to provide an update from the … Rare Pediatric Disease designation from the Food and Drug Administration (FDA). Granted access to the PRIority … data from the study are expected in 2021. Received Orphan Drug designation and Fast Track designation from the FDA. …
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
… has an extensive background in RNA modulation and orphan drug discovery and development and is currently in charge of our R&D. Gerard has … in May 2024. Dr. Shannon has had an extensive career in drugdevelopment and pharma. From 2012 until his retirement …
