Ionis Pharmaceuticals, Inc.

This study tests a new drug called ION717 for people with early-stage prion disease, a rare and fatal brain condition. The drug is given via a spinal injection and aims to reduce harmful prion proteins. The trial will check safety, side effects, and how the drug moves through the…

This program provides access to an investigational drug called donidalorsen for people with hereditary angioedema (HAE), a genetic condition that causes sudden, painful swelling in various body parts. The drug is given as a monthly or every-other-month injection under the skin. I…

This Phase 3 trial tests whether zilganersen (ION373) can improve or stabilize motor function in people with Alexander disease, a rare genetic brain disorder. Fifty-four participants aged 2 to 65 will receive either the drug or a placebo injected into the spine. The study measure…

This study tests a drug called ION582 for people with Angelman syndrome, a genetic condition causing developmental delays and seizures. The drug is given via a spinal injection. The main goal is to check safety and how the body processes the drug. Up to 70 participants aged 0 to …

This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection. This i…

This study is checking the long-term safety of a drug called eplontersen in people with ATTR-CM, a heart condition caused by abnormal protein buildup. About 1400 adults who completed earlier studies will receive eplontersen injections. Researchers will monitor side effects, kidne…

This study tests the long-term safety and effectiveness of donidalorsen, a drug given as a shot under the skin, to prevent attacks of hereditary angioedema (HAE). HAE causes sudden, painful swelling in various body parts. The trial involves 154 participants who previously complet…

This study tests the safety of a new drug, olezarsen, in 24 adults with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fats. All participants have previously taken volanesorsen. The study monitors side effects like low platelet counts and k…

This study is for people with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fats. It tests a drug called olezarsen, given as a shot under the skin, to see if it can lower triglycerides over months to years. Participants are 60 patients who…

This study looks at the long-term safety and effects of a drug called eplontersen in people with a rare inherited nerve disease called hATTR-PN. About 151 participants who completed earlier studies will receive regular injections of eplontersen. The goal is to see if the drug rem…

This study tests the long-term safety of a drug called olezarsen in people with severely high triglycerides, a condition that raises the risk of pancreatitis and heart disease. About 885 participants who completed earlier studies will receive olezarsen injections for up to three …