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ProQR Therapeutics (NASDAQ: PRQR ) announces positive topline results from a Phase 1/2 clinical trial evaluating sepofarsen (QR-110) in patients with Leber congenital amaurosis type 10 (LCA10) due to a certain mutation in the CEP290 gene. More news on: ProQR Therapeutics N.V., Healthca...
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 in the ongoing Phase 2/3 p...
Gainers: Five Prime Therapeutics (NASDAQ: FPRX ) +19% . Ocugen (NASDAQ: OCGN ) +19% . Navios Maritime Holdings (NYSE: NM ) +16% . RISE Education Cayman (NASDAQ: REDU ) +15% . The National Security Group (NASDAQ: NSEC ) +15% . Catabasis Pharmaceuticals (NASDAQ: CATB ) +15% . ele...
LEIDEN, the Netherlands & CAMBRIDGE, Mass., Oct. 07, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced that mana...
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LEIDEN, Netherlands and CAMBRIDGE, Mass., Sept. 30, 2019 (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 announc...
More evil gets done in the name of righteousness than any other way .”― Glen Cook, Dreams of Steel Today we look at a small developmental or ' Tier 4 ' biotech stock from across the pond. The stock is down substantially from levels a year ago but does have multiple 'shots ...
The FDA designates ProQR Therapeutics N.V.'s ( PRQR +2.3% ) QR-1123 for Fast Track review for the treatment of autosomal-dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin (RHO) gene. More news on: ProQR Therapeutics N.V., Healthcare stocks news, Stocks on the move...
LEIDEN, Netherlands and CAMBRIDGE, Mass., Sept. 09, 2019 (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 announc...
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Preclinical proof of concept data for the AX-0810 program targeting NTCP for cholestatic diseases presented at ASGCT 27 th Annual Meeting – Management to host webinar today at 8:00 am EDT to highlight data Martin Maier, PhD nominated to Board with Annual General Meeting scheduled f...
ProQR scientists report for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) leading to meaningful changes in biomarkers in NHPs using Axiomer™ RNA Editing Oligonucleotides Preclinical proof of concept for the Company’s AX-0810 pr...
LEIDEN, Netherlands & CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the An...