Innovator. Advocate.Together with patients, on a mission to advance transformative treatments for people living with serious and rare diseases.
At Akcea, we’re on a mission to transform the treatment of unaddressed or under-addressed serious and rare diseases with leading-edge, RNA-targeted medicines. Whether you’re a patient living with a serious condition, or a physician whose patient desperately needs a therapy, we know you’re counting on us, and it fuels our unwavering commitment to be a partner to the community through innovation.
Cardiometabolic Lipid Disorders and Volanesorsen
There is a significant need for new therapies targeting the protein and lipid risk factors associated with these serious, complex diseases. Our registrational-stage therapy volanesorsen has been developed for two of these diseases: FCS and FPL.
Hereditary Transthyretin (TTR) Amyloidosis and Inotersen
TTR amyloidosis (ATTR) is a rare, progressive and fatal genetic disease that currently only has limited treatment options that do not treat the underlying cause of disease. Inotersen for hATTR amyloidosis is currently under regulatory review for marketing authorization by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).
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Learning about hereditary transthyretin (TTR) amyloidosis
TTR amyloidosis (ATTR) is a rare, progressive and fatal genetic disease that currently only has limited treatment options that do not treat the underlying cause of disease.Visit hattrchangethecourse.com
Learning about familial chylomicronemia syndrome (FCS)
FCS is a severe and rare disease caused by an enzyme deficiency that leads to the buildup of chylomicrons and a high risk of life-threatening pancreatitisVisit FCSFocus.com