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Resources for Patients

What is GNE Myopathy / HIBM?


GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), is a rare inherited disease that causes severe muscle weakness in the legs and arms, usually leading to physical disability within twenty years of onset. Although, some patients are able to live using just a cane for many years, in most patients the quadriceps muscles are spared. GNE Myopathy does not affect the vital organs such as the heart, the lungs and the swallowing ability. 

The disease is found in many populations worldwide: Europeans, South Americans, Africans, Middle Easterns; Arabs, Jews, Persians, and Asians of Japanese and Indian descent among others.

For a more comprehensive description on GNE Myopathy please refer to "GNE Myopathy in Focus", another program devoted to educating patients, families and doctors.

Here is a link to a short video explaining GNE Myopathy

Please see below for the latest Ultragenyx "GNE Myopathy Research Updates for Patients" in multiple languages.

For the most recent editions of newsletters.  Some are translated in multiple languages.