Is there any treatment for Canavan disease?
Canavan disease causes progressive brain atrophy. There is no cure, nor is there a standard course of treatment. Treatment is symptomatic and supportive.
What is the prognosis for Canavan disease?
The prognosis for Canavan disease is poor. Death usually occurs before age 10, although some children may survive into their teens and twenties.
What research is being done on Canavan disease?
The gene for Canavan disease has been located. Many laboratories offer prenatal screening for this disorder to populations at risk. Scientists have developed animal models for this disease and are using the models to test potential therapeutic strategies. Three strategies are currently under investigation: gene transfer to the brain in order to replace the mutated gene for the enzyme; metabolic therapy to provide a crucial missing metabolite (acetate); and enzyme therapy where the enzyme aspartoacylase is engineered to be able to enter the brain and is injected in the the blood stream. Encouraging results have been obtained using these strategies.
Medically reviewed by Jon Glass, MD; American board of Psychiatry and Neurology
"NINDS Canavan Disease Information Page." National Institute of Neurological Disorders and Stroke. 13 June 2013.