Frequently Asked Questions

What is the difference between a registry and a natural history study?

Answer: You can complete the registry in the privacy of your own home. You do not need to go to a doctor's appointment in order to participate. A natural history study requires visits to a study doctor which may include travel.

Are there other clinical trials currently being conducted for GNEM?

Answer: Yes, please see clinicaltrials.gov.

Are there other GNEM registries?

Answer: Yes. There is a national registry for people with GNEM living in Japan through Remudy. The Japanese GNE myopathy registry was set-up in 2012 and is operated by Dr. Madoka Mori from the National Center of Neurology and Psychiatry, Tokyo.

Can I access my health information in the GNEM-DMP database?

Answer: The data protection act grants you the right to access your own health information and to correct anything that may need to be corrected. In the future, you will also have access to your health information by downloading and printing your health information, and have the ability to create reports.

How will I benefit from registering in the GNEM-DMP?

Answer: The registry is intended as a public service for the benefit of people living with GNEM. You will not receive any payment or any other financial benefit as a result of submitting your health information to the registry. The results of research facilitated by the registry may be patentable or may have commercial potential. However, you will not receive patent rights and will not receive financial benefits from any future commercial development.

Nevertheless, there may be other benefits from participating, including the following: if you want, TREAT-NMD will inform you (on the basis of the information that you and your doctor provide) if you might be a suitable candidate for a certain clinical study or trial. If you want, TREAT-NMD will also inform you if any new information on your disease is received which might be of interest to you. The health information collected might also provide benefits for other people with GNEM, for example, by providing information on how many people worldwide have the same disease, or providing information for researchers interested in the best standards of care for GNEM. It will also help raise awareness of the disease and could encourage additional research and development.

What are the potential risks of being in the GNEM-DMP?

Answer: The registry will be stored in a secure database online. Registering over the internet may pose some risk to the security of your health information; however, significant efforts have been put in place to ensure that the security of your information is not breached. You do not give up any of your legal rights by signing this form. There will be no experimental study drug given and no tests or procedures will be performed in the registry; therefore, there are no potential risks or side effects of participating.

How can I update my health information in the GNEM-DMP database if something has changed?

Answer: If there are major changes in the information you provided, we ask that you keep us informed. For example, if your contact information (like your email address) changes or if your medical condition changes, please let TREAT-NMD know by emailing HIBM@treat-nmd.eu.

What happens if I do not want to answer some of the questions or do not know the answer to a question on the form in the GNEM-DMP database?

Answer: You can complete as many questions as you want and are not required to answer every question to participate. You can also ask your doctor to help in answering some questions. You are encouraged to complete as many questions as possible, but you can take a break and come back to complete the questions at a later time.

Is it my decision to participate in the GNEM-DMP and can I withdraw if I change my mind?

Answer: Yes, your participation is voluntary. Even if you decide now to participate, you can change your mind at any time, and there will be no consequences for leaving the GNEM-DMP. The data protection act grants you the right to access your own health information and to correct the health information at any time. Should you wish to withdraw your health information from the GNEM-DMP, you will need to email HIBM@treat-nmd.eu. You will get the same medical care that you receive now whether you participate or not.

If it is in your best interest, or if the GNEM-DMP is stopped, the study supporters may end your participation in the GNEM-DMP without your permission at any time. You will be notified of this decision.

Will the questionnaires on the GNEM-DMP database be available in languages other than English?

Answer: Currently the GNEM-DMP database is available in English. The patient information and informed consent forms are available in the following languages: English, Arabic, French, Hebrew, Italian, Spanish, Cantonese, Korean, and Farsi. If there is a language that you prefer, other than those listed, please email HIBM@treat-nmd.eu for an update on the status of the translation.

Is there testing available to find out if I have GNEM?

Answer: Genetic testing is available for people with symptoms to help diagnosis their medical condition. Please email HIBM@treat-nmd.eu for more information.

Can I ask someone a question not listed here?

Answer: Yes. Please email your question to HIBM@treat-nmd.eu.

Are there other websites with news about GNEM?
Answer: Yes. Please see: