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… to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today … retinal degenerations, autoimmune retinopathy, and gene therapy associated uveitis. He is a principal investigator …

… to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today … in 2021. “From previous clinical trials we have seen that RNA therapies can be a promising approach for patients with … excited to have started a clinical trial for the third RNA therapy in our inherited retinal disease pipeline.” About the …

… to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare … lead to blindness in early childhood. No form of therapy is currently available,” said Dr. Katarina Stingl of … Tübingen, Investigator in the ILLUMINATE trial. “ProQR’s RNA therapy for the p.Cys998X mutation showed the potential …

… to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare … first studies of its kind exploring the impact of ProQR’s RNA therapies on patients with Usher syndrome due to an Exon … STELLAR trial will explore whether QR-421a (ProQR’s RNA therapy) can slow disease progression or even reverse it. …

…     “QR-010 has the potential to be an innovative RNA therapy for patients with CF due to the F508del …

… ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in … to changing lives through the creation of transformative RNA therapies, today announced several presentations at the …

… to changing lives through the creation of transformative RNA therapies for genetic eye diseases, today announced its … Sepofarsen Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital …

… precision medicines based on molecular diagnostics and our RNA therapeutic platform,” said David M. Rodman, MD, Chief … in a planned series of ophthalmology trials utilizing our RNA therapy platform to target the underlying cause of blindness …

… to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today … in 2019 as we advanced our portfolio of investigational RNA therapies for patients that suffer from inherited retinal … Ionis’ proprietary antisense technology. The therapy aims to inhibit the formation of the mutated toxic …