Ionis Pharmaceuticals (formerly known as Isis Pharmaceuticals) is a company that discovers and develops RNA-targeted therapeutics. It provides an antisense technology platform designed to treat complicated and rare diseases. The company offers SPINRAZA, a treatment for children and adults with spinal muscular atrophy (SMA), TEGSEDI, a medicine that treats the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis, and Waylivra, a medicine used to treat familial chylomicronaemia syndrome (FCS).