The Neuromuscular Disease Foundation

The Neuromuscular Disease Foundation has been an invaluable source of support for me since I had to leave work on disability. They provided me with a welcoming and compassionate community that made me feel like I was no longer alone in my journey. This newfound sense of belonging has inspired me to share information about the Neuromuscular Disease Foundation with others, so they too can find the same support and hope that has been instrumental in my life.

This organization and everyone involved seem to geniunely care about the patients and the families. They have helped us tremedously and try to help everyone that has this condition. Wonderful people!!

The Neuromuscular Disease Foundation (NDF) the global leader in research funding exploring therapeutic approaches for the treatment of GNE myopathy *GNEM). The NDF also creates an exchange of information between investigator interested in GNEM to help share resources and combine efforts to enhance the quality of the lives of people living with GNEM through funding critical research focused on treatments. This is exemplified by NDF spearheading and funding the International Gene Therapy Development Program (IGTDP) that is accelerating the development of a gene therapy to treat GNEM.

Incredible! When I was diagnosed with GNE Myopathy my Mom was very scarred. From a Google search she found NDF and called - Lele, the imediante past Executive Director - spent over 90 minutes talking to my Mom, explaining things in non-medical terms that eased here fears. I became involved and truly feel a community that I can talk openingly with others going through what I am. In terms of the org. Structure - they keep expenses crazy low so every penny possible can go into research - research that is paving the way for for treatments and a cure. They care and remain so focused on making patients lives better.

Since January 2022, I have had the great honor to serve as the Executive Director of the Neuromuscular Disease Foundation.

Before joining NDF, I had limited knowledge of rare diseases and didn't know anothing about GNEM.

in the past 10 months, I have not only learned a great deal about GNEM, but have been amazed by the dedication of the scientists with whom NDF works, all around the world, to advance research to help patients.

Likewise, I have talked to patients everywhere, NDF has a presence in more than 50 countries, and intend to do everything in my power the enhance their lives whilst we wait for a cure.

I have to add that NDF has the most remarkable board members, volunteers and patient advocates all of whom are dedicated to helping those with GNEM.

For all these reasons, I know that NDF is the leader in all aspects of scientific development and helping patients.

Excellent organization that is committed to helping fund research to find effective treatment and ultimately a cure for GNEM. NDF is also committed to helping patients and families who live with this disease. They provide a wide range of supportive services and meet patients were they are at in their journey of living, adjusting and coping with their disease.

As a patient living with the relentless fear and anxiety that comes with this ultra rare , progressively debilitating orphan disease, I find my hope in the NDF.

There is so much uncertainty that surrounds this disease, but the one certainty is that the NDF is always there for us- continuing to invest time, money and research into figuring out how to best serve us and our community.

The NDF offers support groups, educational programs, clinics, and genetic testing among so much more and it spreads awareness of our disease in ways that we don’t always have the energy and resources to.

Living with GNEM would be a lot scarier and far lonelier if the NDF didn’t exist. I am so incredibly grateful for all of the work that they do…

NDF has managed to create a community of patients, scientists and donors all over the world with the common goal of supporting , educating and advocating for its patient community while doing everything possible to find a cure for GNE Myopathy.

NDF is doing amazing work, despite this disease being extremely rare. It's able to bring it to conscience of doctors and organizations, fund research to a cure, that don't currently exists, arrange events for patients to help them handle the progression and more!

My name is Eitan Yosefi and I am a patient in GNE myopathy, with hope and expectation that the organization will help us all

The organization enhance the patients well-being and develop a true hope for a cure

The NDF is a great non-profit organization that offers programs and services to rare disease patients: something that is hard to find. They are currently working to find treatment and cure for GNE Myopathy patients.