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Frequently asked questions

Is this a one-time registration process?

Yes, you will only have to register once. However you will be asked to keep your information up to date to ensure that it accurately reflects your current condition and that we can always contact you. You will be asked at least once a year to log back into your account and update your information.

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How long does it take to register?

It will take you about 10-15 minutes to register and provide us with the required information. You can have a look at the questions that are asked before you register.

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How do I know if I have registered successfully?

If you have successfully registered then you will receive an automated email to welcome you to the Global FKRP Registry.  This will indicate the email address that you used to register with.

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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.

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I have forgotten my password. What should I do?

If you forget your password, please follow the instructions on the “Forgot password” page.

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Why do I have to enter dates as year-month-day?

The internationally agreed date format is used to avoid any confusion with defining the correct date. Different countries use different formats and this is designed to reduce the confusion.

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Who enters my medical data?

Part of the medical information is entered by you (or a patient’s parent/guardian if they are under 18 years of age) and the other part is entered by the doctor responsible for the care of your condition (usually a neurologist). The information to be entered by you includes contact details (name, age, address, email, phone number), diagnosis, motor function, pain, ventilation and family history. The information to be entered by your doctor includes presenting age and symptoms, lung, heart and cognitive function, contractures, six minute walk distance and muscle strength. You can see the type of information that will be requested in the example questionnaire(s) in the Information for Patients section.

You will be able to see all of the information collected about you every time you log into your account. You won’t be able to change any of the information that has been entered by your doctor but you can discuss it with them next time you see them during a regular clinic visit if you disagree with what they have entered.

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How do I know if my doctor has filled in my medical data?

Once logged in you will be able to see not only the data that you are asked to enter but also the data that your doctor will be asked to enter. You will not be able to change any of this information but it will be visible to you.

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What can I do if my doctor hasn't entered my medical data or if I feel that it is not correct?

If your doctor has not entered any information about you then you can ask them to do so when you next see them at your regular clinic appointment. If your doctor has entered data but you feel that it is not accurate then this may also be a good opportunity for you to discuss this with the doctor.

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How often should my data be updated?

You should update your data at least annually and an automated email will be sent to you to remind you to do this. You will have access to your account and so you can in fact log in and review the data stored on you at any time and also make any updates as and when it is necessary.

If you move house, change your email address, or get a new phone number we would ask that you make sure you update this information in the Registry so that we are always able to contact you.

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Where will my data be stored?

In the questionnaire we ask you for some personal data and some information about your condition. The information that you enter will be stored in the Global FKRP Registry which is supervised by a Steering Committee. Your data will be stored securely and no unauthorized people will be able to gain access to any information about you.

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Who will have access to my data?

When planning clinical trials, researchers can request information from the Registry about participants potentially eligible for the trial, based on the patients’ clinical and genetic data. Only researchers who have been approved by their local ethics committee and the Global FKRP Registry Steering Committee are allowed access to information held in the Registry.

In the Registry, your data will only be identified by an anonymous code, not by your name. This means that when researchers are provided access to registry data they will not be able to find out your personal information (name, address, etc.), but only the information they need to know about your condition that will help them decide whether you might be suitable for the trial. If they think that you meet the criteria and might benefit from the trial, they will contact the person in charge of the Registry. Staff working for the registry will “de-code” the data to find out the personal details and will contact you to give you information about the trial or about any other issues relevant to your condition. They will not give your name or personal information to the researchers.

In anonymous form, valuable medical data from the registry will be made available to selected researchers around the world, thereby accelerating the research into FKRP mutations and their possible treatments. Additionally, with the advent of clinical trials for some neuromuscular conditions, patient registries mean that patients who may be eligible for certain clinical trials are readily identifiable.

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What happens if I am potentially eligible for a clinical trial?

If you meet the criteria for a particular trial based on the data held about you in the Registry you will be informed about it and provided with some information. If you are interested in the information that you receive about a particular clinical trial, you will be given details to contact the researchers running the trial. If you decide to take part in the trial, you will need to review and sign a separate consent form. You are completely free to make your own decision about any trial we inform you about. If you decide not to take part in a particular trial, your data will still be kept in the registry and we will continue to inform you about other trials unless you tell us not to. Please note that if we tell you about the existence of a trial, this does not imply that we endorse it.

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I want to take part in a clinical trial. If I register, is this guaranteed?

Although one of the main aims of this Registry is to make it easier for people with FKRP-related conditions to be recruited for trials, there is no guarantee that registering your details will ensure that you will be involved in a clinical trial. If you are interested in receiving details of trials that you may be eligible for, please tick the box at the end of the online consent form.
However, it is important that you understand that even if the coordinators of a clinical trial believe that you might be eligible for that trial, based on the data stored about you in the Registry, it is possible that at a later date it will turn out that you do not meet the inclusion criteria (a set of conditions that must be met in order to participate) for the trial after all.

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I do not want to take part in a clinical trial. Should I still register?

We hope that you will be interested in registering even if you do not want to take part in a clinical trial. Your information will still be useful to researchers who are trying to find out more information about people living with FKRP-related conditions, and we will still provide you with other information that might be relevant to your condition. If you do not want to receive any information about clinical trials that you might be eligible for, please tick “no” at the end of the online consent form.

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Who will have access to my medical records?

Staff in charge of the Registry might need to gain access to your medical records to obtain information necessary to the project (for example we will need to ask your geneticist/physician for a copy of your genetic report and also information on your respiratory and cardiac function).

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How will I be identified in the registry?

Your personal details (name, address, etc.) have to be stored in the registry so that we can contact you if we need to inform you about possible clinical trials or anything else that might be relevant to your condition.

This data will be stored in a secure manner and your records will be assigned a unique code. Your records will only be identified by this unique code. Researchers searching the information held within the Registry cannot identify you personally from the information they have access to. Only the registry heads (Prof Volker Straub and Maggie C Walter) and persons explicitly appointed by them will be able to “de-code” the data to get access to your personal details.

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Will my data be kept confidential?

Your data will be kept for an indefinite period at Munich, under the responsibility of Prof Maggie Walter.

Creating a registry requires the existence of a file containing a patient’s personal and medical data. This file will be subject to the regulations on data protection (national laws related to EU directive 95/46). All information we receive from you will be treated confidentially. The information will be encrypted and stored on a secure server.

Third parties wishing to have access to the data in the registry (such as researchers or companies planning clinical trials or conducting research on new therapies) will only have access to anonymous information identifiable by a code. Before they are granted access even to this anonymous information, they have to have permission from an ethics committee. Your data will not be made available to employers, government organisations, insurance companies or educational institutions, nor to your spouse, other members of your family or your family doctor. 

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Can I withdraw and have my data erased if I change my mind?

Your participation in the Registry is completely voluntary. The data protection act grants you the right to access your own data and to rectify them or withdraw them completely at any time. Should you wish to withdraw your data from the registry you will be free to do so without having to provide any explanation. If you wish to withdraw, you should get in touch with the staff in charge of the Registry.

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What if I don’t have a genetic diagnosis?

It is important that you have a genetic diagnosis, not only to be considered potentially eligible for clinical trials but to ensure that you are receiving the most appropriate care for you condition. You should speak to your doctor if you would like to get a genetic diagnosis or if you would like to discuss your diagnosis.

Patients living in the USA may be eligible to receive sponsorship to cover the cost of the genetic test. Please contact the LGMD2I Research Fund for further information.

LGMD Subtyping Tool

There is a free online tool to help guide doctors toward the most probable diagnosis for patients who fall into the LGMD category, called the Automated LGMD Diagnostic Assistant (ALDA). This tool predicts the most likely type(s) of LGMD a patient may have based on clinical presentation and laboratory findings.

If you are a patient interested in using the LGMD subtyping diagnostic tool, please contact either your doctor or the LGMD2I Research Fund to help guide you through the process.

Click here to use the LGMD Subtyping Tool.

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What if I want some more information about patient registries?

If you have more general questions about patient registries you may like to view the “Patient Registries - Questions and Answers” section of the TREAT-NMD Alliance website.

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