Canavan disease

Clinical Research/Studies

Is there clinical research happening on Canavan disease?

The website is a U.S. government website that provides regularly updated information about federally and privately supported clinical trials. This is not an exhaustive list of current research.Current studies can found by searching for 'Canavan disease' on

In Europe, the EU Clinical Trial Register performs a similar service.

To learn about clinical trials sponsored by private sources, you can contact

In addition to the clinical research studies, there is ongoing research into gene therapy, enzyme replacement therapy and stem cell therapy. Gene therapy seeks to replace the defective gene in a person with an unaffected one that can produce the necessary aspartoacylase enzyme. Enzyme replacement therapy replaces the aspartoacylase enzyme that is deficient in Canavan disease with an artificial version that is created in a lab and injected into a person with this disorder. As of 2019, gene therapy has not been approved by the U.S. Food and Drug Administration for the treatment of any disorder. Enzyme replacement therapy has been approved by the FDA for the treatment of disorders that are similar to Canavan disease. website.

NINDS Canavan Disease Information Page. National Institute of Neurological Disorders and Stroke website.

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