Neurol Genet 2021 Apr 9;7(2):e572. Epub 2021 Mar 9.
Department of Neurology (S.W., K.G., B.S.), Friedrich-Baur-Institute, Ludwig-Maximilians-Universität München, Germany; Institute of Molecular, Cell and Systems Biology (S.A.C., D.G.M.), University of Glasgow, United Kingdom; Department of Neurology (K.O., B.G.M.v.E.), Radboud University, Nijmegen, The Netherlands; Institute of Genetic Medicine (A.C.J.-M.), Institute for Ageing and Health, Newcastle University, United Kingdom; Neuromuscular Reference Centre (F.D., G.B.), Assistance Publique-Hôpitaux de Paris, France; Department of Neuropediatrics and Muscle Disorders (H.L.), University of Freiburg, Breisgau, Germany; Center for Genomic Regulation (H.L.), Barcelona Institute of Science and Technology, Spain; Tayside Clinical Trials Unit (F.H.), The University of Dundee, United Kingdom; and Department of Medical Psychology (H.K.), Amsterdam University Medical Centers, University of Amsterdam, Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.
Objective: To assess the association between variant repeat (VR) interruptions in patients with myotonic dystrophy type 1 (DM1) and clinical symptoms and outcome measures after cognitive behavioral therapy (CBT) intervention.
Methods: Adult patients with DM1 were recruited within the OPTIMISTIC trial (NCT02118779). Disease-related history, current clinical symptoms and comorbidities, functional assessments, and disease- and health-related questionnaires were obtained at baseline and after 5 and 10 months. Read More