Ionis Pharmaceuticals, Inc.

This study is testing a drug called donidalorsen in children aged 2 to 12 who have hereditary angioedema (HAE), a rare condition that causes sudden, severe swelling. The main goal is to see if the drug is safe and well-tolerated in young children, and to measure how it works in t…

This early-stage study is testing a new drug called ION717 for people with prion disease, a rare and fatal brain condition. The drug is injected into the spinal fluid to try to lower the harmful protein that causes the disease. Researchers will check if the treatment is safe and …

This is the first study in people to test the safety and side effects of an experimental drug called ION464 for multiple system atrophy (MSA), a rare and serious brain disorder. About 40 adults with MSA will receive the drug or a placebo via a spinal injection to see how their bo…

This study is testing whether the experimental drug ION582 can improve symptoms in children and adults with Angelman syndrome, a rare genetic disorder that affects development. About 158 participants aged 2 to 50 will receive either the drug or a placebo for about 60 weeks, follo…

This study is testing a new drug called ION582 for Angelman syndrome, a rare genetic disorder. The drug is given as an injection into the spinal fluid. The main goals are to see if it's safe and how the body processes it in about 70 participants, including children under 2 years …

This study is the first to test a new drug called ION356 in children with Pelizaeus-Merzbacher disease (PMD), a rare genetic disorder that affects the brain and nerves. The main goal is to check if the drug is safe and how the body processes it when given as a spinal injection. A…

This study is testing a new drug called ION440 for people with MECP2 Duplication Syndrome, a rare genetic disorder. The main goal is to see if the drug is safe and how the body handles it. The drug is given via a spinal injection, and the study will include both children and adul…

This study aims to better understand how Pelizaeus-Merzbacher disease (PMD) progresses over time in children. Researchers will collect spinal fluid, blood samples, and brain scans from up to 32 boys with PMD over about two years. The information gathered will help identify biolog…