Information for Researchers

The Registry supports and encourages applications from researchers working to improve knowledge, understanding, care and treatment of myotonic dystrophy. Research which may benefit from interactions with the registry includes but is not limited to:

  • Clinical research that will improve standards of care and best practices
  • Scientific research that increases understanding of disease mechanism and pathology
  • Epidemiological studies to better understand the prevalence and progression of the condition.
  • Clinical trials of new therapies and treatments.

The registry is a flexible research tool and can facilitate and accelerate research in a number of ways:

  • Feasibility enquiries which provide non identifiable aggregate data to help you plan and carry out your research, e.g. the number of patients with a specific symptom or from a specific region.
  • Facilitating the recruitment process, the registry can directly contact patients fitting specific criteria to provide information about participating in your research.
  • Providing services to conduct research including disseminating, collecting and analysing research questionnaires.

It is important to note that the Registry releases de-identified data only; patient identifiable data will never be released to a researcher, company or other third party.

If you are interested in using the registry then contact the registry team as soon as possible to discuss further how the interaction could be best used to aide your research. Early contact will ensure that all approvals are met and all costs are negotiated.

Fee Structure

The UK Myotonic Dystrophy Patient Registry is supported by charitable funding from patient organisations in the United Kingdom. The registry desires to help all researchers, academic or commercial to improve knowledge, understanding and treatment of the condition. As a result, the Registry charges fees on a cost-recovery basis. The Registry encourages all parties interested to contact the team regarding the interaction as early as possible. Early contact helps to facilitate project planning and delivery and may afford the opportunity to amend grant applications and update project budgets where applicable.

Application Process

Any research wishing to use the registry must be submitted to the Registry Steering Committee. This board is made up of doctors, scientists and patient organisation representatives. Data will only be released after approval is given by Registry Steering Committee. It is not within the remit of the registry to provide ethical or scientific review of research but the Committee may ask to see evidence that such review has been carried out by the appropriate bodies.

The amount of information needed varies depending upon the scope of request please contact us for more information.

International research

If you are interested in conducting research internationally then please contact TREAT-NMD (info@treat-nmd.eu). All myotonic dystrophy registries share the same core dataset allowing them to act as a single resource for information and research. TREAT-NMD is experienced in coordinating registries on global level (www.treat-nmd.eu/registries).