Editorial Comment: Brown-Vialetto-Van Laere and the Advances in Molecular Medicine

Brown-Vialetto-Van Laere syndrome is just one example of a disease known for over a century with no effective treatment where the recent identification of the gene defect brings to light the possibility of new therapeutic targets. Fundamental to the rational approach to therapy is the understanding of the genetic and or molecular pathophysiology of a disease. Once this is understood, even to a limited degree, a number of targets for potential therapeutic intervention can be identified. Usually these are targets, which would not be intuitive to the physician otherwise.

Source: Seminars in Pediatric Neurology - April 19, 2017 Category: Neurology Authors: John B. Bodensteiner Source Type: research