Information for Researchers

The registry exists not only as a source of information but importantly to help facilitate and accelerate your research into FSHD. There are a number of ways the registry can help to do this;

• Planning research: The registry can help you identify not only the number of patients but also the location of these patients in the UK, discovering a potential cohort beyond the large neuromuscular centres. In addition we can provide de-identified aggregate data about specific aspects of the condition which can be invaluable in assessing the feasibility of a potential study.
• Recruiting for research: The registry can contact large numbers of patients who have indicated they are willing to take part in clinical research; making them aware of recruitment periods and letting them know about your research. We can target those members who best meet the eligibility criteria.
• Conducting research: The registry can be used to conduct research, through questionnaire based services, helping with analysis

 

The Registry supports and encourages applications from any researcher, academic or industry, working to improve knowledge, understanding, care and treatment of FSHD. 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

• Pre-clinical 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.

 

Application Process

Any research proposal including the Registry must be submitted to the Registry Steering Committee for approval. This board is made up of doctors, scientists and patient organisation representatives (more details). 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.

Every enquiry is different and the amount of information needed varies depending upon the scope of request so please contact us for more information.

The list of questions each registry participant is asked to answer can be viewed here and the additional clinical questions provided by doctors can be found here.
 

Fee Structure

The UK FSHD Patient Registry is entirely supported by charitable funding from the Muscular Dystrophy UK. As a result fees are associated with industry use of its services for feasibility, recruitment and research purposes. These fees are dependent on the work being carried out and we would encourage any parties interested in using the registry to contact us as early as possible to discuss this. Early contact helps to facilitate project planning and delivery and may also afford the opportunity to update the project budget where applicable.

 

International research

For multi-national studies the TREAT-NMD Alliance (treat-nmd.org) can help unite and organise the many national FSHD registries they are affiliated with which all collect the same dataset. Please contact TREAT-NMD ([email protected]) for more information about Global Registry Enquiries.