Ionis Pharmaceuticals, Inc.

This study tests a new medicine called ION337 in 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 an…

This Phase 3 study tests an experimental drug called ION582 in 158 children and adults with Angelman syndrome, a genetic disorder causing severe developmental delays. The drug is given as a spinal injection and aims to improve expressive communication and cognitive skills. Partic…

This phase 2 trial tests an injectable drug called ION775 in 72 adults with high or severely high triglycerides. The goal is to see if it safely lowers fasting triglyceride levels. Participants continue standard care while receiving the study drug.

This study tests a drug 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 how it works in young bodies. Researchers will monitor side effects and measure h…

This early-stage trial tests an experimental drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare and progressive brain disorder. The drug is given via a spinal injection and aims to reduce levels of a protein linked to the disease. The main goal is to check…

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 study tests a new drug called ION440 in 48 people with MECP2 duplication syndrome, a rare genetic condition that causes intellectual disability and seizures. The drug is given via a spinal injection to see if it is safe and how the body processes it. Some participants will r…

This program offers early access to zilganersen for people with Alexander disease, a rare genetic brain disorder. It is for US residents aged 2 and older who have not responded to or cannot tolerate approved treatments. The goal is to provide the drug while it is still being stud…

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 check if the drug is safe and how the body processes it. The drug is given through a spinal injection to target …

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 observational study is following 32 boys aged 6 months to 17 years with Pelizaeus-Merzbacher disease (PMD), a rare genetic disorder affecting the brain's white matter. Researchers are measuring biomarkers in spinal fluid and blood, brain scans, and motor and cognitive skills…