There is currently no treatment available for any aspect of the NARS1 disease and there is an overwhelming need to develop a treatment of this lethal condition. Some patients on anti-epileptic treatment (Keppra) have seen control over their generalized seizures.
Gene therapy is a relatively new treatment technology that holds immense potential for treating patients with such genetic diseases. We aim to work on a mouse model, which will help us develop an experimental treatment for this disease. This trial will move research forward in developing a potentially disease-modifying treatment for NARS1 disease, providing hope to individuals and families where currently none exists.
NARS1