Conclusions

Abstract

The key to forming a comprehensive understanding of clinical neurology is an appreciation for the basic science elements of neuronal functioning at the cellular and molecular level. Similarly, therapeutic advances build upon and rely on a dual understanding of the clinical and scientific aspects of neurologic disease. Current examples of previously untreatable fatal neurologic diseases that are now on the cusp of a major therapeutic advance include Duchenne muscular dystrophy, with clinical trials now exploring exon skipping so that functional proteins of abbreviated length can be “stitched together” and still preserve protective levels of dystrophin. New basic science imaging methods using optogenetics and fluorescent antibodies promise new insights to brain functioning.