Thirty-four clinicians, scientists and representatives from industries convened for a workshop on cerebral involvement in myotonic dystrophy (DM). The workshop was held in Ferrere (Asti) from May 24th to 27th 2013 and as the previous one [1]had the purpose to stimulate the research on CNS dysfunction in DM and to discuss major issues regarding CNS involvement.
Classically viewed as a neuromuscular disease, for many years research on DM has been principally focused on muscular aspects. Therefore few data are available for CNS involvement. With new therapeutic developments with potential to affect pathophysiology across multiple tissues and organ systems it will be mandatory to increase our understanding of CNS pathophysiology in order to appropriately monitor whether or not there is beneficial effect in CNS. Indeed CNS dysfunction is one of the major issue affecting quality of life in DM patients thus it has to be appropriately considered in planning clinical trials.
This workshop included different themes covering all the topics of DM CNS involvement. CNS research needs to proceed faster and consensus has to be established in the major themes of CNS study. The present report describes the major issues that emerged during the workshop, with the aim of updating and stimulating research in this critical field
Neuromuscular Disorders 2014
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