What advances in upstream medical therapies inform neurorestoratology

Abstract

Recent advances in upstream medical therapies for neuromuscular disorders suggest that the best outcomes result from their administration in the pre-symptomatic, and perhaps, neonatal period. Currently available therapies, and many other extremely expensive therapies in the pipeline soon to be considered by the Food and Drug Administration, and suggest the importance of avoiding potential life-threatening disease complications for patients to continue to benefit from these. There is evidence that this is almost always possible with the use of respiratory muscle aids to avoid pneumonias and respiratory failure, but these are currently little understood and rarely offered by the medical community. However, restoring neuromuscular function necessitates keeping the patient alive and well.