We work to enhance the quality of life for those living with GNE Myopathy by funding critical research focused on treatments and a cure through The International Gene Therapy Development Program (IGTDP) and by advocacy, education and outreach.
We work to enhance the quality of life for those living with GNE Myopathy by funding critical research focused on treatments and a cure through The International Gene Therapy Development Program (IGTDP) and by advocacy, education and outreach.
Our Virtual Learning Library provides recordings of our GNEM Speaker Series events, podcasts, and webinars and is an invaluable resource where you can find answers to many of your questions about GNE Myopathy and the work of NDF.
Our impact at a glance:
50+
Our Global Impact
Leif
Switzerland
"The hardest thing for me is to accept it and it's still difficult to show myself with it in public.
Everybody is vain. In addition, it is the uncertainty of how far the disease will progress.
Realizing in one morning that something that worked a short time ago doesn't work today."
Osamu
Japan
"I was born in Japan, raised in Japan, and live in Japan. I did a muscle biopsy, genetic testing, etc. Then, a few years later, I also did genetic testing of my parents. So, GNE myopathy was confirmed. I have depression as a complication. And I keep taking my medicine. I go to the hospital regularly. I currently use a reclining electric wheelchair."