Is there clinical research happening on Canavan disease?
As of May 2016, there are several clinical research studies for Canavan disease listed on clinicaltrials.gov, 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 are looking at specific treatment options and genetic carrier testing. More information can be found by searching for ‘Canavan disease’ on ClinicalTrials.gov.
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 May 2016, 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.
SOURCE: Emory University - Department of Human Genetics in collaboration with ThinkGenetic • https://www.thinkgenetic.com/diseases/canavan-disease/clinical-research/8510 • DATE UPDATED: 2016-05-08
NINDS Canavan Disease Information Page. National Institute of Neurological Disorders and Stroke website. http://www.ninds.nih.gov/disorders/canavan/canavan.htm