Data access

The UK SMA Patient Registry aims to facilitate and accelerate research into spinal muscular atrophy (SMA). There are a number of ways we can do this:

  • Planning research: The patient registry can provide information on the number and the regional location of affected individuals across the UK and Ireland, unlocking a potential cohort beyond the large neuromuscular centres. In addition, we can provide de-identified data about specific features of the condition which can be invaluable in assessing the feasibility of a potential study.
  • Recruiting for research: The patient registry can assist study recruitment by contacting individuals with SMA, against certain eligibility criteria if required, to inform them about the research and recruitment pathways of universities, the NHS or companies.
  • Conducting research: The patient 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 patient 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 SMA.

Application Process

Any request to use the UK SMA Patient Registry must be reviewed and approved by the independent patient registry Steering Committee. This board is made up of doctors, physiotherapists 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 Steering 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 patient 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.

Additional Access

The UK SMA Patient Registry is a member of the TREAT-NMD SMA Global Registries Network and therefore, with the approval of the patient registry Steering Committee, is able to contribute to enquiries into this network.

The UK SMA Patient Registry collaborates with clinical networks SMA REACH UK (children) and with Adult SMA REACH. With patient consent, the patient-reported outcome measures (PROMs) captured in the patient registry are shared with the SMA REACH studies to supplement reports to UK regulatory authorities. This important information will help inform regulatory authorities in their decision of whether NHS funding of a treatment should continue.