Ionis Pharmaceuticals, Inc.

This early-stage study tests a new drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare brain disease that affects movement and balance. The main goal is to check if the drug is safe and tolerable when given as a spinal injection. Researchers will also measu…

This study tests a new medicine called ION337 in 32 children aged 2 to 12 with Dravet syndrome, a severe form of epilepsy. The main goal is to see if the drug is safe and tolerable when given as a spinal injection. Researchers will also measure how the drug moves through the body…

This study tests an experimental drug called ION775 in 72 adults with high or very high triglycerides, a type of fat in the blood that can raise heart risk. Participants must already be on standard cholesterol-lowering medicines. The main goal is to see if ION775 safely reduces t…

This early-stage study tests a new medicine called ION356 in 24 boys aged 2 to 17 with Pelizaeus-Merzbacher disease, a rare genetic brain disorder. The main goal is to see if the drug is safe and tolerable when given via spinal injection. Researchers will also measure how the dru…

This program offers zilganersen to people with Alexander disease, a rare genetic condition that damages the nervous system. It is for US residents aged 2 and older who have not responded to or cannot tolerate other treatments. The goal is to provide access to this experimental dr…

This study tests a medicine called donidalorsen in 20 children aged 2 to 12 with hereditary angioedema (HAE), a condition that causes sudden, painful swelling. The goal is to see if the drug is safe and helps reduce the number of swelling attacks. Participants receive the drug an…

This study tests a new medicine called ION440 for people with MECP2 duplication syndrome, a rare genetic condition that causes intellectual disability and movement problems. The drug is given via a spinal injection to see if it is safe and how the body processes it. About 48 male…

This study tests a new drug called ION717 in 76 people with early-stage prion disease, a rare and fatal brain condition. The drug is given via a spinal injection to see if it is safe and how the body processes it. The goal is to slow or stop the disease, but participants will lik…

This study tests an experimental drug called ION582 in 70 people with Angelman syndrome, a genetic condition causing developmental delays and seizures. The drug is given via spinal injection to try to improve symptoms. The main goal is to check safety and how the body processes t…

This study tests a new medicine called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The goal is to see if the drug can improve communication and thinking skills. Participants will receive the drug or a placebo, …

This study follows 32 boys and young men with Pelizaeus-Merzbacher disease, a rare genetic brain disorder, to measure how the disease changes over time. Researchers will collect spinal fluid, blood, and brain scans, and track movement, thinking, and daily function. The goal is to…