Akcea Therapeutics

A rare autosomal recessive disorder, Familial chylomicronemia syndrome (FCS) affects 1 in 1 to 2 million people (Burnett and Hegele, 1999; Pouwels et al. 2008). As per DelveInsight, the total Familial chylomicronemia syndrome diagnosed prevalent population in the 7MM [the US, EU4 (Germany, Franc...

FDA approves transthyretin amyloidosis therapy of Akcea, Ionis Patients with an orphan, hereditary disease that is transthyretin amyloidosis with a second new treatment option are available after the Food and Drug Administration approved Akcea Therapeutics and Ionis Pharmaceuticals' Tegsedi.  As Tegsedi is approved,...