- A total of 27 participants including molecular biologists, clinicians, geneticists, and patient advocates from 13 countries convened from the October 11–13, 2019 in Hoofdorp, The Netherlands, for the 248th ENMC International Workshop, on the topic Myotonic dystrophies: molecular approaches for clinical purposes, framing a European molecular research network.
- An ENMC workshop to develop a European consortium for care and therapy for myotonic dystrophy (DM) was held on 1st to 3rd of July 2016 in Naarden, the Netherlands. Twenty-five participants from nine countries participated. The main objectives were to build on the knowledge gained and tools developed from the observational prolonged trial in myotonic dystrophy type 1 to improve quality of life standards, a target identification collaboration (OPTIMISTIC) study and to bring additional expertise from outside the consortium with a particular focus on outcome measures and registries.
- In September 2014, twenty-two experts in the field of Pompe disease from nine European countries gathered in Naarden, The Netherlands, for a workshop on Pompe disease. Experts were mostly physicians with clinical experience in treating and following larger groups of patients with Pompe disease and in doing research on this disease. Additionally, an epidemiologist, a basic scientist and a patient representative were present. The aim of the workshop was three-fold:
- The therapeutic strategies for myotonic dystrophy 2010 workshop was held in Banff, Alberta, Canada, from September 8–10, 2010. This meeting was the second, biennial therapeutic strategy workshop sponsored by the Marigold Foundation and was attended by 52 participants spanning academic and clinical investigators with an interest in myotonic dystrophy (DM), industry participants with an interest in neuromuscular disease and/or rare disorders, and representatives from regulatory and funding agencies.
- A workshop entitled Patient Registries and Trial Readiness in Myotonic Dystrophy, jointly sponsored by TREAT-NMD ( www.treat-nmd.eu ) and the Marigold Foundation ( www.marigoldfoundation.org ), was held from 12 to 14 June 2009 in Naarden, The Netherlands. The twenty-six participants represented eight countries and included scientists, clinicians, patient representatives and industry. The workshop built on the foundations established in two previous ENMC workshops on myotonic dystrophy and the myotonic dystrophy clinical working group set up by the Marigold Foundation, and took advantage of the tools developed within the TREAT-NMD network for patient registries and outcome measures.