PELIZAEUS-MERZBACHER DISEASE

This early-stage study tests a new drug called ION356 in 24 boys aged 2 to 17 with Pelizaeus-Merzbacher disease, a rare genetic disorder affecting the brain. The main goal is to check if the drug is safe and how the body processes it. The drug is given via a spinal injection to t…

This early-stage study tests whether adding special cells (DUOC-01) to a standard cord blood transplant is safe for children with inherited metabolic diseases that damage the brain. About 40 participants, from 1 week to 21 years old, will receive the cells via a spinal injection.…

This study collects medical information and biological samples from up to 12,000 people with leukodystrophies—rare brain disorders affecting white matter. Researchers will use this data to find new genetic causes, develop biomarkers, and better understand how these diseases progr…

This study follows 1,500 people with rare genetic brain diseases to learn how these conditions progress. Researchers will track thinking, movement, and daily living skills over time. The goal is to better understand the diseases and how treatments might affect them.

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 …