Ionis Pharmaceuticals is engaged in RNA-targeted therapeuty. Co.'s commercial products are SPINRAZA, TEGSEDI and WAYLIVRA. SPINRAZA injection for intrathecal use is a survival motor neuron-2 directed antisense medicine indicated for the treatment of spinal muscular atrophy in pediatric and adult patients. TEGSEDI injection is an RNA-targeted medicine indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. WAYLIVRA is an antisense medicine indicated as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.
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