New hope for treatment of rare metabolic disease

Results of a controlled clinical trial with a new drug for the treatment of X-linked adrenoleukodystrophy To date, no pharmacological treatment exists for X-linked adrenoleukodystrophy, a progressive genetic metabolic disease that damages the adrenal glands, the spinal cord and the white matter of the nervous system. A new drug currently in the approval process could help prevent severe disease progression, which is usually fatal. This is indicated by the results of the world's first controlled clinical trial on this rare, genetic metabolic disease. Scientists at Leipzig University Hospital, together with an international research team from the United States and Europe, have published their important findings in the prestigious journal -The Lancet Neurology-. X-linked adrenoleukodystrophy (X-ALD) is the most common of a group of around 50 rare diseases of the white matter of the brain, the so-called leukodystrophies. The genetic damage in X-ALD is due to a defect in the X chromosome. Affected men suffer from progressive paralysis of the legs, deterioration of balance and sensory disturbances, and develop incontinence and sexual dysfunction.