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Published on: May 12, 2023
A randomized controlled study of modified cobratoxin in adrenomyeloneuropathy
H R Mundy1, S J Jones, J C Hobart
1Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK. helen.mundy@uclh.org
Abstract:
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.
