238th ENMC International Workshop: Updating management recommendations of cardiac dystrophinopathyHoofddorp, The Netherlands, 30 November – 2 December 2018

Twenty four neuromuscular experts, cardiologists,
patient representatives and a trainee, supported by the
ENMC Young Scientist Programme, from seven European
countries (Belgium, Czech Republic, France, Germany,
Italy, Netherlands, United Kingdom) and the United States
participated in the workshop, held in Hoofddorp, the
Netherlands. The aims of were to: (1) agree diagnostic
standards, thresholds for initiating therapy and an optimal
therapy regime for cardiac dystrophinopathy; (2) determine
whether there are clinically relevant genotype-phenotype
correlations for cardiomyopathy in DMD; (3) review current
knowledge on the potential of genetic therapies to prevent
cardiac dystrophinopathy and (4) agree on guidance for
the use of implantable cardioverer defibrillators and left
ventricular assist devices in the management of patients with
advanced cardiomyopathy