There are many types of neurological diseases that affect infants and children. Although the frequency of individual disorders is not high, together they are a significant group of disorders with a collective frequency of 1 in 18,000 live births. Unfortunately, neurologic disease is seldom curable. Thus, strategies for the treatment of these debilitating and often fatal diseases frequently focus primarily on palliative measures. Attempts at curing neurological disease have also been proposed. These treatments have included enzyme replacement therapy, gene therapy, and allogenic bone marrow transplantation. Sadly, however, these treatments typically do not improve the condition nor alter the ultimate outcome of the disease, leaving a desperate need to develop effective therapies.