Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study
Patients who have a particular genetic disease whose symptoms include progressively worsening muscle control were able to walk faster following treatment with an experimental Ionis Pharmaceuticals genetic medicine, meeting the main goal of a pivotal study. Based on these results, the drugmaker said it plans a regulatory submission for what could become the first FDA-approved…