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 …