We are recruiting participants (12 years and older) in a clinical trial for USH2A mediated Usher syndrome and Retinitis Pigmentosa. Check if you're eligible!
ProQR is recruiting children under 8 years old with Leber congenital amaurosis for a clinical study of an investigational RNA therapy. Learn more about the study and how to participate.
See the ongoing and planned clinical trials and how you can contribute to research. Contact us for more information or for clinical trial participation.
ProQR is conducting a pivotal clinical study of an investigational RNA therapy for people with Leber congenital amaurosis (LCA10). Learn more about the study and the sepofarsen program.
Clinical trials help researchers and doctors learn more about genetic eye diseases and new medications. Read about participation, safety and how to join a clinical trial.
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.
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
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.
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.
