Study Suggests New Strategy for Treating Rare Neurodegenerative Disorder Menkes DiseaseReleased December 1, 2015
- Menkes disease arises from dysfunction in ATP7A, a protein that transports copper to cells, leading to brain development complications. Introducing working versions of ATP7A in the brain is considered the most direct therapeutic approach. However, a new study in AJP-Cell suggests that functioning ATP7A located elsewhere in the body, not necessarily the entire brain, can help treat the disorder. This research is highlighted as one of this month’s “best of the best” as part of the American Physiological Society’s APSselect program.