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About NDF

History & Mission


History:

Founded in 2006 by Gila Mikail, NDF is a registered 501(c)(3) public charity, and the world's leading foundation in funding research that supports the search for a cure for GNE Myopathy (also known as HIBM). Research is just one of the four programs within the NDF. Click here to learn more about these programs.The organization is governed by its rotating Board of Directors, managed by its Executive Director, and supported by volunteer committees, including a distinguished Scientific Advisory Committee

Our Mission Statement:

The Neuromuscular Disease Foundation's (NDF) mission is to serve the GNE Myopathy/HIBM community through patient advocacy, raising awareness, educating physicians and the general public, and funding clinical research in search of a cure for neuromuscular diseases.

Achievements in 2015:

  1. NDF has funded over $1 million to researchers and scientists in the field of HIBM/GNE Myopathy worldwide. Grants have been made through organizations such as Hadassah Medical Center in Israel and UCLA, and now these efforts have been expanded to include sample collection and genetic sequencing in countries and towns in the Middle East, such as Iran and Uzbekistan, known to have a higher than average rate of HIBM carriers. These samples provide a unique opportunity to learn more about the disease in the quest for a cure.
  2. Although the origin and cause of most rare diseases is not known, with the help of scientists, NDF understands more about the genomes causing the disease, and has not only one, but two organizations working on the final stages of their treatments in clinical trials
  3. NDF has established collaborations with the National Institute of Health (NIH) as well as Ultragenyx, both of whom have dedicated resources and millions of dollars to developing a treatment for HIBM patients.
  4. The prevention and suppression of HIBM in future patients is a significant part of NDF's mission. Therefore, our focus on screening carriers with not only the hope for early detection, but also the knowledge that we can prevent the passing on of the mutations through science.
  5. Exciting new developments in science, such as CRISPR Cas 9, genome editing, offers us hope for the ability to edit human genes. Out ability to test this type of gene therapy, first by making the mouse-models healthy, can be eventually transferred over to human beings, through a process that essentially allows scientists to "cut and replace" affected DNA with healthy DNA.