This observational study aims to understand how Alexander Disease, a condition affecting the brain's white matter (leukodystrophy), changes over time. Researchers will collect information on how the disease impacts your movement, speech, swallowing, and overall quality of life. They will also collect blood and spinal fluid (cerebrospinal fluid or CSF) samples to measure levels of a protein called GFAP. This information will help design future studies to test new treatments for Alexander Disease. You can join if you have a diagnosis of Alexander Disease. The study is looking for 200 participants, but its current recruitment status is unclear.

Study design

This is an observational study, meaning researchers will watch how the disease progresses without testing a specific intervention. It aims to enroll 200 participants.

What's involved

You will have physical exams, physical therapy, occupational therapy, speech and language therapy, brain function, and swallowing assessments. You or your caregiver may also fill out questionnaires, and optional specimen collection is available. You will be asked to return at least once a year for repeat assessments.

Compensation

Not stated in the trial record.

Follow-up

Your gross motor function will be measured for up to 10 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.