Data access

This registry aims to facilitate and accelerate research into myotubular and centronuclear myopathies. There are a number of ways we can do this;

  • Planning research: The registry can provide information on the number and the location of affected individuals across the world, unlocking a potential cohort beyond the large neuromuscular centres. In addition we can provide de-identified aggregate data about specific aspects of the conditions which can be invaluable in assessing the feasibility of a potential study.
  • Recruiting for research: The registry can contact affected individuals, against certain eligibility criteria if required, to inform them about your research and recruitment pathways.
  • Conducting research: The registry can be used to conduct research through stand-alone online questionnaires, and can help with analysis and reporting if required.

If you are interested in using the registry please contact us as soon as possible to ensure adequate time for scoping and approvals. We encourage applications from any patient, researcher, academic or industry stakeholders working to improve knowledge, understanding, care and treatment of MTM or CNM.

Application Process

Any request to use the registry must be reviewed and approved by the independent Registry Steering Committee. This board is made up of doctors, scientists and patient organisation representatives and their role is to ensure that registry data is used appropriately and safely. It is not within the remit of the Committee to provide ethical or scientific review of research but they 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.

Fee Structure

To support registry sustainability and cover the cost of conducting the required work, a fee may be charged for use of registry data. We encourage interested parties to contact us as early as possible in their planning stages, as early contact may afford the opportunity to amend grant applications and update project budgets where applicable.