(Coordinator: Baziel van Engelen, Nijmegen, Netherlands): an observational prolonged trial in myotonic dystrophy type 1 to improve QoL standards, a target identification collaboration.

Submitted under: HEALTH.2012.2.4.4-2: Observational trials in rare diseases

The aim of OPTIMISTIC is to improve clinical practice in the management of patients with Myotonic dystrophy for which no dedicated treatment is currently available. To this end OPTIMISTIC compares two intervention regimes (exercise training and cognitive behavioural therapy (CBT)) and evaluates their effectiveness and adverse events, with particular attention given to the definition of appropriate outcome measures and new clinical guidelines for DM1 management. Relevant stakeholders such as national and European patient organisations are engaged and rapid uptake of developed guidelines is ensured by dissemination plans involving these stakeholders as well as the international TREAT-NMD network and other DM1 specific networks.

Project length - 3 years
Project award - up to €3million
12 Apr 2017