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What is Canavan Disease and How is It Treated?

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Canavan disease treatment and description Hemera/Thinkstock

In terms of treatment, there is no standard course, and it tends to target the symptoms of Canavan disease, according to the National Institute of Neurological Disorders and Stroke.

But a new preliminary study may have found a possible treatment for Canavan disease. The researchers used a gene therapy method that was developed at the University of North Carolina School of Medicine.

It involves taking a normal copy of the ASPA gene and putting it into a non-infectious virus. It is then directly infused in patients’ brains. This gene therapy reduces the levels of NAA and slowed neuronal degeneration.

The study by Leone et al. treated a total of 13 children, all of whom are still alive about a decade later.

The gene therapy resulted in small improvements in symptoms, such as muscle rigidity and ability to move. It also reduced the number of seizures in some of the children.

In the beginning of the study, the gene therapy was administered to children who were 5 years old on average. HealthDay News noted that this group showed the least amount of improvement.

As the study progressed, gene therapy was allowed on younger participants — 16 months old on average — who displayed the most benefit from the treatment.

While this treatment is showing promise for patients with Canavan disease, it is still experimental and not approved as a treatment in the United States. In addition, there is not good natural history data on this disease.


Genetics Home Reference. Canavan Disease. Web. 24 December 2012.

National Institute of Neurological Disorders and Stroke. Canavan Disease Information Page. Web. 24 December 2012.

MedlinePlus Medical Encyclopedia. Canavan Disease. Web. 24 December 2012.

Leone, P. et al. (19 December 2012). “Long-Term Follow-Up After Gene Therapy for Canavan Disease.” Science Translational Medicine. Web. 24 December 2012.

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We value and respect our HERWriters' experiences, but everyone is different. Many of our writers are speaking from personal experience, and what's worked for them may not work for you. Their articles are not a substitute for medical advice, although we hope you can gain knowledge from their insight.


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