Ionis Pharmaceuticals, Inc.

This study looks at the long-term safety of a drug called eplontersen in people with a rare inherited nerve disease (hATTR-PN). About 151 participants who completed earlier studies will receive the drug for an extended period. The main goal is to check for side effects and monito…

This study looks at the long-term safety of a drug called eplontersen in people with a heart condition known as ATTR-CM, where abnormal proteins build up in the heart. About 1400 adults who completed earlier studies or have this diagnosis will receive the drug and be monitored fo…

This study tests a medicine called donidalorsen for people with hereditary angioedema (HAE), a condition that causes sudden, painful swelling. The goal is to see if the drug is safe and effective over the long term at preventing these attacks and improving quality of life. About …

This study looks at how well the drug olezarsen works over time to lower fat levels in the blood of people with familial chylomicronemia syndrome (FCS), a rare genetic condition. About 60 participants who completed a prior study will receive the drug for up to 3 years. The main g…

This study is for people with a heart condition called ATTR-CM, where a protein called transthyretin builds up in the heart. It tests whether the drug eplontersen can reduce that buildup compared to a placebo. About 150 participants will have special heart scans at 140 weeks to s…

This study tests a new drug called olezarsen in 24 adults with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fats. Participants have previously taken volanesorsen. The main goal is to check safety, especially effects on blood cells and org…

This study looks at the long-term safety of a drug called olezarsen for people with severe hypertriglyceridemia (very high blood fats). About 885 adults who finished a prior olezarsen study will receive the drug and be monitored for side effects and lab changes over up to 3 years…

This study tests an experimental drug called zilganersen (ION373) in 54 people with Alexander disease, a rare brain disorder. The drug is given via spinal injection and aims to improve or stabilize walking and other motor functions. Participants are aged 2 to 65 and must have a c…

This study tests a new drug called sapablursen in about 50 people with polycythemia vera, a rare blood cancer that causes too many red blood cells. The goal is to see if the drug can reduce how often patients need phlebotomy (removing blood) and improve their symptoms. Participan…

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, and th…

This study uses MRI scans to measure amyloid protein buildup in the heart of up to 150 people with a heart condition called ATTR-CM. Participants are already in a larger treatment study. The goal is to see how the amount of amyloid changes over time, not to test a new treatment. …