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Frequently Asked Questions

Where can I get more information about GNE myopathy?

Genetics Home Reference:

Advancement of Research for Myopathies (ARM):

Neuromuscular Disease Foundation (NDF):

Where will I have to go to participate in the study?

You will be admitted to the NIH Clinical center for this study.

Will I get paid to participate in this study?

You will not receive any direct compensation for your participation.

Will I have to pay for my travel expenses to the clinical study center?

Travel expenses will be paid by the NIH. For international patients, we will pay travel from point-of-entry to the US.

Can I bring a companion? Would his/her travel will be paid for?

Yes, we will pay for a companion if it is medically indicated.

What are the benefits to participating in this study?

By participating in this study, you will help to improve our understanding of GNE myopathy, which is an important contribution to families and individuals affected by this condition. This study will provide information that will help all clinical trials that explore new therapies for GNE myopathy.

Does participating in the GNE myopathy Natural History Study mean that I am automatically enrolled in the ManNAc trial?

No it does not. The eligibility criteria for the ManNAc trial might be different from those for this natural history study.

Why should I stop taking ManNAc while I participate in this study?

We need to know your muscle status at baseline (i.e., without ManNAc) and would like to compare it with your muscle status when you were receiving ManNAc.

I have multiple family members with HIBM, can we all participate?

Yes! As long as each person has evidence of a diagnosis of GNE myopathy and signs informed consent, you may all participate. Furthermore, we can coordinate to bring you and your siblings or friends to the study at the same time.

How do we participate?

You simply need to fill out the form for participation (see link on left navigation bar) and email the form to us. You may also email or call us to enroll.

Is there a method that is preferred for me to fill out the form?

The preferred method is the method that is most convenient for you. We are happy to accommodate you via phone, hard copy, or computer.

Will anyone know that I took part in this study?

Only those directly involved in this research at NIH will know of your participation in this study.

Where will the study take place?

At the National Institutes of Health Clinical Center in Bethesda, MD. For more information, visit or what to expect from your visit.

What will you do with the information you learn about me and/or my family who participate in this study?

We will analyze this information in a composite fashion, with no names attached to the report.