Open AccessClinical Outcome Evaluations and CBT Response Prediction in Myotonic Dystrophy
Author(s) -
D. van As,
Kees Okkersen,
Guillaume Bassez,
Benedikt Schöser,
Hanns Lochmüller,
Jeffrey C. Glen,
Hans Knoop,
B.G.M. van Engelen,
Peter A.C. ’t Hoen
Publication year - 2021
Publication title -
journal of neuromuscular diseases
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.366
H-Index - 23
eISSN - 2214-3602
pISSN - 2214-3599
DOI - 10.3233/jnd-210634
Subject(s) - myotonic dystrophy , medicine , outcome (game theory) , physical medicine and rehabilitation , pediatrics , mathematical economics , mathematics
The European OPTIMISTIC clinical trial has demonstrated a significant, yet heterogenous effect of Cognitive Behavioural Therapy (CBT) for Myotonic Dystrophy type 1 (DM1) patients. One of its remaining aims was the assessment of efficacy and adequacy of clinical outcome measures, including the relatively novel primary trial outcome, the DM1-Activ-c questionnaire.