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GNE Myopathy (HIBM) Patient Information Form

If you are a diagnosed GNEM patient and would like to be included in our Patient Registry, please click below to fill out our Patient Information Form and submit it to us via email at or fax it to (310)-496-0199.

Prefer to complete your Patient Registry form online? Click here.


NDF Patient Registry FAQs:


What is a patient registry?

It is a collection of standardized information about individual persons who share a condition which is collected in a systematic and comprehensive way and can serve a variety of purposes.

Shared informational data allows for a deeper and broader understanding of the nature of a disease and its patient population.

Who is eligible?

Anyone, from any region of the world, who has been diagnosed with GNE Myopathy eligible to participate.

Is there a cost to register?

There is no cost to register.

Why should I share my data?

By sharing your data, you are contributing to real scientific research, empowering yourself with updates shared with registered patients, adding to the collective power of the patient community and helping NDF build its case with industry professionals for their investment in our mission.

Why is NDF collecting this information?

o    Collecting information about as many GNEM patients as possible helps us determine and predict the number of identified cases of GNEM worldwide in support of research and potential biotech industry partners.

o    Collecting information helps our scientific collaborators increase their understanding of the patient population and the disease, support design for potential natural history studies and clinical trials for new treatments, assist with the development of recommendations for standards of care and characterize the GNEM population as a whole.

o    Having contact information for patients allows NDF to be in contact with patients about important and worthwhile programming, studies and trials specifically tailored to their community.

o    Learning more about where GNEM patients live helps us identify regions where more attention is needed.

Is my data secure and who will have access to my information?

NDF is the guardian of the information contained in the Registry. Only NDF staff and program managers who are part of the registry team have access to the secure data center that contains personal information.

Patients’ information is de-identified when shared with any qualifying NDF scientific collaborator. Your name and contact information will never be given to anyone without your permission.  NDF is committed to protecting your privacy. 

When requested and warranted for specific studies, NDF may share this information (de-identified) with scientific collaborators who request this information to move their studies forward. These requests are approved by NDF’s Registry Advisory Committee, which is comprised of GNEM research experts.

Information included in NDF registry will never be sold.

I’ve participated in GNEM/HIBM registries in the past, how is this different?

NDF is the largest nonprofit organization in the world working specifically on GNE myopathy (HIBM). As such, it is vital that we continue in our efforts to build the most comprehensive database of patients ever created. Potential researchers and biotech companies require data (always de-identified) in order to invest time, energy and resources into any potential work on any rare disease.  Our registry is an important component in our efforts to continue building our impressive coalition of experts working on this disease.

Does my doctor need to be involved in my participation?

While we may ask who your doctor is in order to be able to share this information with another patient living in your area in need of a referral, there is no requirement to have a doctor involved in your participation.

Should you have any further questions about the NDF registry or your participation in it, please contact us at