Ionis Pharmaceuticals, Inc.

This study is checking the long-term safety of an experimental drug called olezarsen for people with Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder that causes extremely high fat levels in the blood and painful pancreas attacks. It is for 24 people who have alre…

This study is testing whether a new drug called ION373 can help improve or stabilize movement and motor function in people with Alexander disease, a rare genetic brain disorder. About 54 patients aged 2-65 will receive either the drug or a placebo for 60 weeks, followed by the dr…

This study provides continued access to the experimental drug olezarsen for up to 60 patients with Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder causing extremely high blood fats. The main goal is to gather long-term safety data and see if the drug keeps loweri…

This study is testing an experimental drug called ION363 for people with ALS caused by specific mutations in the FUS gene. The drug is delivered via spinal injection and aims to slow the loss of physical function and improve survival. The trial will involve about 95 participants …

This study uses heart MRI scans to see if an experimental drug called ION-682884 can reduce the buildup of a harmful protein (amyloid) in the heart muscle. It involves up to 150 people who already have a heart condition caused by this protein buildup, known as ATTR-CM. The goal i…

This study is testing the long-term safety and effectiveness of donidalorsen, a medication designed to prevent swelling attacks in people with hereditary angioedema (HAE). The research involves 154 participants who will receive the treatment for up to two years while researchers …

This study follows patients who previously participated in earlier trials of eplontersen, a medication for hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), a rare nerve disease. Researchers want to see if the drug remains safe and effective when taken for up t…

This study aims to check the long-term safety of a medication called eplontersen for people with a serious heart condition called ATTR-CM. About 1,400 patients who completed a previous study will receive the drug by injection every 4 weeks for up to 3 years. Researchers will moni…

This study uses special heart scans to see if an investigational drug, eplontersen, can reduce the buildup of a harmful protein in the heart. It involves up to 150 people who already have a heart condition called ATTR amyloidosis and are part of a larger drug trial. The main goal…

This study is testing whether a drug called sapablursen can help people with a blood disorder called polycythemia vera. The main goal is to see if the drug reduces how often patients need blood drawn to manage their condition and if it improves their quality of life. About 50 par…

This study aims to check the long-term safety of the drug olezarsen in people with severely high triglycerides. It follows about 800 patients from two earlier trials for up to 3 years of treatment. The main goal is to see if the drug causes any side effects or health problems ove…