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.
Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
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.
