ION356 clinical trials

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

This study is looking at a new treatment called ION356 for boys aged 2-17 with Pelizaeus-Merzbacher Disease (PMD). It aims to see how safe the treatment is and how the boy's body handles it. It's an early-stage study to understand more about this potential new medicine.