GNE Myopathy is a rare inherited disease that causes progressive muscle weakness. The disease is caused by defects of the GNE gene. In this disorder the defect of the GNE enzyme disrupts the functioning of the sialic acid metabolic pathway, and interferes with normal muscle function.  Read More >>

Gene therapy is a promising treatment option for genetic diseases that have no cure. Gene therapy is used to introduce a normal copy of the gene to restore function to cells. Gene therapy uses a vector, typically a virus that does not cause disease, to deliver the gene to cells in the body.  Read More >>