Testing a new spinal injection treatment for Lafora disease

This is a Phase 1/2 clinical trial testing a new treatment called ION283, which is injected directly into the fluid surrounding the spinal cord. The trial aims to see if this medication is safe and whether it can help reduce symptoms in teenagers (ages 10–18) who have been diagnosed with Lafora disease, a rare genetic condition that causes progressive seizures and movement problems.

Lafora disease is a serious inherited epilepsy with no cure, and patients gradually lose the ability to control seizures and move independently. This trial exists to explore whether this new medication, delivered directly to the nervous system, might slow disease progression or improve quality of life.