What is Alexander disease?
Alexander disease is one of a group of neurological conditions known as the leukodystrophies. Leukodystrophies are disorders that result from abnormalities in myelin, the “white matter” that protects nerve fibers in the brain.
In Alexander disease, the destruction of white matter is accompanied by the formation of Rosenthal fibers—abnormal clumps of protein that accumulate in non-nerve cells (astrocytes) in the brain and causes the nervous system to function abnormally.
Alexander disease can appear at any age, although most cases appear in infancy or early childhood. There is no known cure for the disease, but other symptoms may be treated. The disease is often fatal.
How can I or my loved one help improve care for people with Alexander disease?
Consider participating in a clinical trial so clinicians and scientists can learn more about Alexander disease and the leukodystrophies. Clinical research uses human volunteers to help researchers learn more about a disorder and perhaps find better ways to safely detect, treat, or prevent disease.
All types of volunteers are needed—those who are healthy or may have an illness or disease—of all different ages, sexes, races, and ethnicities to ensure that study results apply to as many people as possible, and that treatments will be safe and effective for everyone who will use them.
For information about participating in clinical research visit NIH Clinical Research Trials and You. Learn about clinical trials at Clinicaltrials.gov.
Where can I find more information about Alexander disease?
The following organizations and resources help individuals, families, friends, and caregivers of people living with Alexander disease:
Genetic and Rare Diseases (GARD) Information Center
National Organization for Rare Disorders (NORD)
Phone: 203-744-0100 or 800-999-6673; 844-259-7178 Spanish
United Leukodystrophy Foundation
Phone: 815-748-3211 or 800-728-5483