LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 04, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S.
… ProQR Receives Fast Track Designation from the FDA for QR-110 … for Leber’s Congenital Amaurosis Type 10 Key Updates ProQR receives Fast Track designation by the U.S. Food and … which there is currently nothing available,” said Noreen R. Henig, Chief Medical Officer of ProQR. “We are also …
Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence
Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well tolerated