GNE

We host an international annual symposium on GNE Myopathy , delivering education and support programs and connecting stakeholders, physicians, scientists, and researchers with community members... We maintain one of the largest GNE Myopathy patient registries in the world to ensure that the community is organized and trial-ready and to collect data needed for better disease understanding... 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.

Also known as: GNEM

Primary location: Beverly Hills United States