INTRODUCTION
The ENMC ensures financial support for workshops, offers organisational support through its office in Baarn, The Netherlands, and is a partner in the development of collaborative activities in the European Neuromuscular Research community including TREAT-NMD. The ENMC stimulates and facilitates top-level scientific collaboration for many neuromuscular diseases including Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Post Polio Syndrome, Limb Girdle Dystrophies, and Congenital Muscular Dystrophies.
Workshops Approximately 8 workshops on neuromuscular disorders are held each year. The aim of these workshops is to encourage and facilitate collaborative research. For example, diagnostic criteria for a number of neuromuscular disorders based on clinical features and laboratory investigations were drawn up and agreed upon at the workshops. The workshops also focussed special attention on locating, isolating, and characterising genes for specific disorders. More recently, a focus on clinical trial development was led by Professor Richard Hughes and Dr Michael Rose.
Applications for grants for the Workshops are based on scientific merit and each application is objectively assessed by the ENMC Research Committee. Applicants base their requests on the Criteria for grant applications. This method of dealing with applications has proved not only easy to manage, but ensures that applications are assessed objectively by internationally recognised experts in the field. Each workshop has the option to establish a Consortium for on-going coordinated research in a particular field under an elected Chairperson.
ENMC Clinical Trial Network
The ENMC funded a Clinical Trial Group led by Professor Richard Hughes and Dr Michael Rose together with the Cochrane Collaboration. Via this trial network, ENMC fostered the development of clinical trials for the treatment of neuromuscular disease. The ENMC will build on previous and future workshops to provide the evidence base for the treatment of neuromuscular disease. This activity is continuing in partnership with TREAT-NMD.
Workshop Support Projects Most ENMC-supported collaborative Workshops and activities share certain objectives such as the development and implementation of diagnostic criteria and collection of epidemiological data. Collaborative groups greatly welcome support, input, and guidance in these areas. These projects run parallel with issues prioritized by the EU.
Clearing House ENMC offers additional support to collaborative Consortia by acting as a clearing house for information coming from and going to members of the respective Consortia. In this way, a central address and clearing house for all relevant scientific and other information is maintained. Additionally ENMC aims to inform the scientific and lay community on current scientific developments within its scope.
Results To date, 172 workshops on a variety of neuromuscular disorders have been organized. At least 116 publications directly related to these Workshops have been published, as well as several indirectly related publications. In total approximately 1500 participants from over 30 countries have been actively involved in these Workshops. Through these participants several hundreds of their colleagues have directly benefitted from the deliberations and results.
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