A Cure for Sanfilippo Syndrome? A Summary of Current Therapeutic Approaches and their Promise

Abstract

The goal of post-acute neurorehabilitation is to maximize patients' function, ideally by using surviving brain and central nervous system tissue when possible. Yet the structures incorporated into neurorehabilitative approaches often differ from this target, which may explain why efficacy of conventional clinical treatments targeting neurological impairments varies widely. Non- invasive brain stimulation such as with Transcranial Magnetic Stimulation (TMS) and transcranial direct current stimulation (tDCS) offers the possibility of directly targeting brain structures to facilitate or inhibit their activity so as to steer neural plasticity in recovery, and measure neuronal output and interactions for evaluating progress. Latest advances as stereotactic navigation and electric field modeling are enabling more precise targeting of patient's residual structures in diagnosis and therapy. Given its promise, this supplement illustrates the wide-ranging significance of TMS and tDCS in neurorehabilitation, including in stroke, pediatrics, traumatic brain injury, focal hand dystonia, neuropathic pain and spinal cord injury. TMS and tDCS are still not widely used and remain poorly understood in neurorehabilitation. Thus, the present supplement includes articles that highlight ready clinical application of these technologies, including their comparative diagnostic capabilities relative to neuroimaging, their therapeutic benefit, their optimal delivery, the stratification of likely responders, and the variable benefits associated with their clinical use due to interactions between pathophysiology and the innate reorganization of the patient's brain. Overall, the supplement concludes that whether provided in isolation or in combination, non- invasive brain stimulation with neuro-rehabilitation are synergistic in the potential to transform clinical practice.