The purpose of the FUSION study is to determine if a potential new treatment, or “investigational antisense medicine,” is safe and effective in halting, reversing, or slowing deterioration of clinical functioning and biomarkers of disease severity in ALS patients with FUS mutations
- Amyotrophic Lateral Sclerosis is a rare neurological disorder that affects the nerve cells in the brain and spinal cord. People with ALS may have trouble with certain muscle movements like speaking, walking, or gripping. About 1-5% of ALS cases are caused by a mutation in the FUS gene, leading to quicker ALS progression beginning at an earlier age.
- The investigational antisense medicine or placebo will be injected into the spinal fluid in the lower spine region and circulate to the spinal cord and brain. ION363 is designed to target and stop the production of the FUS protein in the body. Targeting this protein may improve symptoms by reducing FUS protein buildup in motor neurons.