Ionis Pharmaceuticals Receives FDA Approval for Rare Genetic Disorder Drug

Ionis Pharmaceuticals' drug has been approved by the US FDA for a rare genetic disorder. The drug, which targets a specific RNA, is used to treat patients with spinal muscular atrophy (SMA), a progressive and often fatal genetic disease. Ionis Pharmaceuticals has five marketed medicines and a pipeline in neurology, cardiology, and other areas of high patient need. The company's products include SPINRAZA, QALSODY, WAINUA, TEGSEDI, and WAYLIVRA.