Imagine you are a strong, healthy young adult with a lifetime of promise ahead of you. One day, you notice that you are having trouble bending your index finger. Or you realize you can't hold your foot up the way you used to. Or people start asking you why you're limping. You have no idea what's happening to your body, nor does anyone else. You are scared and confused and realize that you are helpless to stop the slow and terrifying transformation taking place in your body. That is what HIBM feels like. And that's just the beginning.
AWARENESS. SCREENING. CURE.
We are a nonprofit organization whose mission is to raise awareness and encourage testing for HIBM and to find a cure for neuromuscular diseases, including HIBM. Our vision is a world free of HIBM today and for generations to come.
HIBM is a rare and debilitating form of muscular dystrophy that causes young people’s muscles to waste away and robs them of their ability to do all of the everyday activities that the rest of us take for granted. While the recessive form of the disease is most common among Iranian Jews and the Japanese community, HIBM affects people around the world. There is currently no treatment or cure.
With your help, NDF will continue to direct funding to scientists who are working to develop a treatment and cure for HIBM.
A public-private partnership has launched an HIBM natural history study and patient registry.
National Human Genome Research Institute (NHGRI) is recruiting patients for a Natural History Study of patients with HIBM.
Ultragenyx Phase 2 Sialic Acid Study Results Presented at American Academy of Neurology on April 30, 2014