Ionis Pharmaceuticals, Inc.

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 early-stage study tests a new medicine called ION356 for Pelizaeus-Merzbacher disease (PMD), a rare genetic brain disorder. The main goal is to check if the drug is safe and tolerable in 24 boys aged 2 to 17. The drug is given via a spinal injection to target the central ner…

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. The medicine is given as an injection into the spine. Researchers will also measure how the body proc…

This program offers zilganersen to people with Alexander disease, a rare genetic brain disorder, who have not responded to or cannot tolerate approved treatments. It is for US residents aged 2 and older with a confirmed GFAP gene variant. The goal is to provide access to this exp…

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 early-stage study tests an experimental drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare and serious brain disorder. The main goal is to check the drug's safety and how well people tolerate it when given via a spinal injection. Researchers will also…

This study is for boys aged 6 months to 17 years with Pelizaeus-Merzbacher disease (PMD), a rare genetic brain disorder. Researchers will measure changes in spinal fluid and blood markers, brain scans, and motor and thinking skills over time. The goal is to better understand how …