Aceneuramic acid is the first approved drug for GNE myopathy treatment
In order to treat an underserved population of patients with a rare condition whose muscles gradually become weaker until they can no longer walk, a team of researchers across Japan have completed a clinical study to confirm the safety of long-term administration of a therapeutic drug. Distal myopathy with rimmed vacuoles, or GNE myopathy, is … Read more
