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Familial chylomicronaemia syndrome: olezarsen
Familial chylomicronaemia syndrome (FCS) is a rare genetic disease that causes extremely high levels of triglycerides. It is the result of the enzyme lipoprotein lipase not working properly, which leaves patients unable to break down chylomicrons effectively
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Therapeutic: donidalorsen for hereditary angioedema
Hereditary angioedema (HAE) is a rare genetic disease that’s characterised by rapid and painful attacks of inflammation in the limbs, hands, feet, face, larynx, trachea and abdomen. If swelling takes place in the larynx, it can be fatal