Huntington's disease: Therapeutic potential of triheptanoin confirmed
The therapeutic potential of triheptanoin in 10 patients with Huntington's disease has been demonstrated by researchers. Huntington's disease is a genetic disease; mutation of the gene encoding the huntingtin protein results in a progressive degeneration of the neurons, especially in regions of the brain involved in the control of movement, thereby causing serious neurological, motor, cognitive and psychiatric problems. Weight loss is also observed in patients at an early stage in the disease, despite normal or even increased food intake. These two observations (degeneration of neurons and loss of weight) led the researchers to propose the hypothesis that an energy deficit in these patients might play an important role in the onset and progression of the disease symptoms.