UK Patient Registry for Myotonic Dystrophy

General information

Background and purpose

For the treatment of Myotonic Dystrophy, promising new therapeutic strategies are currently being developed which need to be tested in clinical trials/research studies. This registry will make the recruitment of Myotonic Dystrophy patients for trials or studies easier by helping to identify suitable patients for particular trials or studies and by enabling them to be contacted and informed quickly when there is a trial or study they might be interested in. More information

Target group

This registry is for patients living in the United Kingdom who are affected by Myotonic Dystrophy (DM1, Curschmann-Steinert, or DM2, Proximal myotonic myopathy 'PROMM'). This registry is primarily designed to register patients who might be suitable for participation in future clinical trials of new therapies, and to help the researchers find the best way of caring for patients with Myotonic Dystrophy. Therefore, this registry is intended for patients currently living with the condition and not as a record for those who have already died.

Advantages for patients

If you are a patient affected by Myotonic Dystrophy and register here, we can direct you to clinical trials and research results (such as new treatments) that might be of specific interest to you. In addition, by registering you are helping researchers obtain precise data about the prevalence of Myotonic Dystrophy, which could be of benefit to all affected patients. More information

Required data

The required data will be asked for in a two-part questionnaire.

  • Part 1: This part is to be completed by the patient or carer / guardian / parent of the patient. You can view all the questions in advance.
  • Part 2: This part is to be completed by a professional involved, for example by a geneticist or a neurologist/neuromuscular specialist. The information required is related to test results which would not necessarily be readily available to the patient, such as test results related to their respiratory function, cardiac status, mutation analysis and outcome scores.

Registration process

Patients can register on their own online by clicking “Registration”, which leads them to the web application for the UK Myotonic Dystrophy Patient Registry. There is a step-by-step user guide to assist you with the registration process here, and you can also contact the registry curator if you would like any additional support. This is a dual-entry registry, which means that patients initiate the registration themselves and their selected healthcare professional enters additional information if selected.

In order to register, you should first read the appropriate patient information sheet (see below) and agree to register by consenting online. Then you can register by entering your personal and contact details. In the next steps you and your doctor fill in the online questionnaire. In this questionnaire, questions are related to your myotonic dystrophy condition.

If necessary, you can save and interrupt your registration at any time, for instance in order to consult a doctor to discuss questions. More information on the exact registration process.

Patient Information Sheets

The Patient Information Sheets (PIS) can be viewed via the links below. You will be asked to confirm you have read and understood these before completing your consent form, so if you have any questions please contact the registry curator before registering. Please note than patients under the age of 16 must be registered by their parent/guardian who will provide consent on their behalf; the patient will be contacted to reconsent as an adult when they turn 16.

The registry team are working on a new PIS and consent form to allow family members to enter data on behalf of adult patients who do not have the capacity to consent. Please keep an eye on the registry newsletters for details of when this will be launched.

 

Data access

Staff in charge of the registry have access to your data and can contact you if you might be suitable for a particular study. Furthermore, the doctor – who you choose and authorise in the form yourself – will fill in the second part of the questionnaire, and will be able to view the first part of the questionnaire. More information about data protection

Data updates

To make sure that the data in the registry are correct and valuable, it is essential that we update them regularly. To do this, we will send you emails once a year asking you to tell us about any changes in your medical condition. You can easily update your data yourself at any time by logging into the registry by entering your e-mail address and password and changing your data. If there are any major changes in your medical or contact details that occur in the period between the annual updates, please also notify us or update your data yourself. Such changes could be for example a change of address or the loss of ambulation.

Data protection

Your data are stored on a specially secured computer, which can be accessed only by selected people. Information that you enter online via the Internet is encrypted while being transferred, so that it cannot be intercepted. More information about data protection

Help

Here you will find answers to some questions that you might have when using the registry.

I had to temporarily interrupt my registration. When I wanted to continue, I had to type in my password. Why?

For security reasons you are logged out automatically if you have been idle for two hours, i.e. not clicked on a link or button or typed in any text. After you type in your password as requested, in most cases you should be back on the page you were on before you were logged out. If not, you can click through to the desired page via the navigation bar on the left. If at the time you were automatically logged out you had made changes in a form without clicking the “Save” button, you will have to make these changes again.

I have forgotten my password. What should I do?

If you forget your password, please follow the instructions on the “Forgot password” page. In case you can't remember with which e-mail address you have registered, please contact us.

 

If you have any more general questions about patient registries, you may like to view the “Patient Registries - Questions and Answers” resources on the TREAT-NMD Neuromuscular Network website.