… 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 RNAtherapy 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 RNAtherapy 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 RNAtherapy) can slow disease progression or even reverse it. …
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
… 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 …