Ionis Pharmaceuticals, Inc.

Today we reported Q3 2024 financial results. Read more about our updates and tune in live to the webcast today at 11:30 AM ET to hear our third quarter highlights: https://lnkd.in/deQDzcDx

Today, we announced the pivotal Phase 3 study design for our investigational #AngelmanSyndrome (AS) medicine following alignment with the U.S. FDA. Read more: https://lnkd.in/g7azfbZw #RareDisease

The U.S. Food and Drug Administration has accepted our New Drug Application for our investigational RNA-targeted prophylactic medicine for the treatment of hereditary angioedema (#HAE) with a decision date of August 21, 2025.?? ? Read more about this milestone: https://lnkd.in/eW7GxPpk #RareDisease??

For someone #livingwithFCS (familial chylomicronemia syndrome), every meal and social event can be a challenge. Because FCS prevents the body from digesting fats and severely impairs the body’s ability to remove triglycerides from the bloodstream, consuming even small amounts of fat could trigger a trip to the emergency room.? ??? To close out #FCSAwarenessDay, watch the video to better understand how an evening out to dinner can cause anxiety for those who have FCS. To learn more, visit https://lnkd.in/egGqBYsi.??

Familial chylomicronemia syndrome (FCS) is a rare, genetic and potentially life-threatening disease that is characterized by extremely high triglyceride levels. Is the following a misconception or fact?? ? FCS can be fully controlled and managed by eating a low-fat diet.? ? This #FCSAwarenessDay, click the images below to discover the answer and visit https://lnkd.in/dqj6nGs to learn more about #livingwithFCS.?

Today we recognize #FCSAwarenessDay to pause and shine a light on the experience of people #livingwithFCS (familial chylomicronemia syndrome). FCS is a rare, genetic disease for which there are currently no approved therapies in the U.S. Those living with FCS have a high risk of potentially life-threatening acute pancreatitis (painful inflammation of the pancreas) and manage sometimes daily symptoms of fatigue and severe stomach pain. Currently, the only way to manage FCS in the U.S. is with an extremely strict diet and lifestyle adjustments that limit daily fat intake to less than 15 to 20 grams. Take a glimpse at a day with FCS. Learn more: https://lnkd.in/dqj6nGs.

Save the date for our Q3 2024 earnings webcast. Register now and tune in live on November 6 at 11:30 AM ET: https://lnkd.in/e69xq3He

We’re pleased to share that Dr. Tomoyo Hamana is this year’s recipient of our CardioRenal-Metabolic Young Investigator Grant Program! A research fellow at CVPath Institute, Inc., Dr. Hamana will leverage the grant to conduct the first comprehensive examination of the relationship between genetic variants that cause high triglycerides and the specific mechanisms of cardiovascular disease development and progression.

It’s the 4th #WorldAmyloidosisDay, an annual occasion to recognize this group of rare diseases caused by the buildup of abnormal proteins (called amyloids) in tissues and organs throughout the body. Because this condition's signs and symptoms are similar to those of more common diseases, it can take more than three years to receive an accurate #amyloidosis diagnosis. ? ATTR (transthyretin amyloidosis) is a type of amyloidosis caused by buildup of a specific protein called transthyretin (TTR). Given the progressive nature of ATTR amyloidosis, an accurate and timely diagnosis is critical. ? Watch the video to understand the different forms of ATTR, and learn more about this complex disease from Amyloidosis Alliance: https://lnkd.in/e97AZmyK

We’re at the?American College of Allergy, Asthma and Immunology (ACAAI) Annual Scientific Meeting presenting new three-year data from the Phase 2 open-label extension (OLE) study & additional results from our Phase 3 OASIS & OASISplus studies for our investigational RNA-targeted prophylactic medicine for hereditary angioedema (#HAE).? ? Read more:?https://lnkd.in/ectuq7Gg