Over the years, the disorder known today as GNE Myopathy, or GNEM, has gone by many names. If you or your loved one is living with GNEM, then you may have also heard it referred to as:
- Hereditary Inclusion Body Myopathy
- Nonaka Myopathy
- Distal Myopathy with Rimmed Vacuoles
- Inclusion Body Myopathy 2
- Quadriceps-Sparing Myopathy
All of these names have pointed to the same muscular degenerative disease. As the scientific community’s knowledge of this disorder has grown, so the labels for this condition have continued to evolve. Looking forward, there will be a single treatment solution that will halt the progression of GNEM and its symptoms in patients. NDF proudly relies on some of the world’s most elite leaders in clinical research, gene therapy, and drug development – many of whom we are honored to have as members of our own Scientific Advisory Committee (SAC) – to bridge the gap between the past decades of struggle for patients with GNEM and medical practitioners working to treat it, and a future wherein GNEM’s symptoms, adjoining struggles and labels are eradicated by a single effective therapy. We are so close.
If you would like to learn more about the members of our esteemed Scientific Advisory Committee (SAC), data from current and past NDF funded studies, or stay up-to-date with real-time news on the progression of NDF’s IGTDP plan to an effective treatment that will end GNEM, please subscribe below to receive timely updates!