PELIZAEUS-MERZBACHER DISEASE

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 early-stage study tests a new medicine called ION356 in 24 boys aged 2 to 17 with Pelizaeus-Merzbacher disease, a rare genetic brain disorder. The main goal is to see if the drug is safe and tolerable when given via spinal injection. Researchers will also measure how the dru…

This study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…

This study follows 32 boys and young men with Pelizaeus-Merzbacher disease, a rare genetic brain disorder, to measure how the disease changes over time. Researchers will collect spinal fluid, blood, and brain scans, and track movement, thinking, and daily function. The goal is to…

This study follows 1,500 people with rare genetic brain disorders like MLD, Krabbe disease, and Batten disease. Researchers track thinking, movement, and language skills over time, and collect blood and MRI scans. The goal is to learn how these diseases progress, not to test a tr…