The neurological disability created by stiffness and twisting of the muscles is difficult to cure. A comfortable wheelchair is essential. Several medications may be useful, but none has been evaluated in a controlled clinical trial. Frequently used medications include baclofen, diazepam, and clonazepam. These are usually given as pills by mouth, but some get baclofen delivered directly to the spinal cord region by a small pump. The main goal of these medications is to reduce the muscle stiffness and improve mobility.
Severe mental retardation is uncommon in Lesch-Nyhan disease, but most have a significant learning disability. The most serious problem appears to be a very short attention span. But in the proper environment in a special school, they can learn many things.