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Neurology 2003;61:528-530
© 2003 American Academy of Neurology
Brief Communications

A randomized controlled study of modified cobratoxin in adrenomyeloneuropathy

H.R. Mundy, MBBS, S.J. Jones, PhD, J.C. Hobart, PhD, M.G. Hanna, PhD and P.J. Lee, MD

From the Charles Dent Metabolic Unit (Drs. Mundy and Lee), Department of Neurophysiology (Dr. Jones), and Department of Clinical Neurology and Neurorehabilitation (Dr. Hobart), National Hospital for Neurology and Neurosurgery, Queen Square; and Muscle and Neurogenetics Section (Dr. Hanna), Institute of Neurology, Queen Square, London, UK.

Address correspondence and reprint requests to Dr. Mundy, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK; e-mail: helen.mundy{at}uclh.org

Adrenomyeloneuropathy is a peroxisomal disorder that causes demyelination, with no proven therapy. Oral modified cobratoxin was assessed in a double-blind, randomized, crossover study of eight patients. Treatment was well tolerated. There were no significant improvements with therapy. The authors do not confirm previous anecdotal reports of dramatic improvement with modified cobratoxin.