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GNE Myopathy, also known as HIBM, is an adult onset genetic disorder affecting people of all ethnicities, but more prevalent in people of Jewish, Iranian, Indian, Irish, Scottish and Japanese descent. There are currently 2 potential treatments in the works, and NDF is pursuing gene therapy in this area. Genetic screening is encouraged to avoid passing down the gene to future generations.

Dr. Rosenbaum Congratulates NDF on Initiating the Pre-IND for Gene Therapy