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May 1979

Treatment of Duchenne's Muscular Dystrophy With Penicillamine: Results of a Double-Blind Trial

Author Affiliations

From the Departments of Neurology (Drs Roelofs, Saavedra de Arango, and Law), Physical Therapy (Miss Kinsman), Psychiatry (Dr Buchanan), and Physiology (Dr Park), Vanderbilt University School of Medicine, and the Jerry Lewis Neuromuscular Disease Research Center (Drs Roelofs, Saavedra de Arango, Law, and Park), Memphis, Tenn. Dr Roelofs is now at the University of Minnesota, Minneapolis; Dr Saavedra de Arango is now at Hospital Universitario del Valle Cra, Colombia, South America.

Arch Neurol. 1979;36(5):266-268. doi:10.1001/archneur.1979.00500410044005

• Eleven boys with Duchenne's muscular dystrophy, randomly assigned to placebo (group A, n = 6) or penicillamine treatment (group B, n = 5), received three capsules per day containing lactose or 250 mg of penicillamine. All patients received pyridoxine, 50 mg daily. Mean age at entrance into study was similar for both groups (group A, 86.7 ± 31.6 months; group B, 95.4 ± 43.4 months). Clinical status was assessed with timed functional activities, manometric measurements of muscle force, and manual muscle testing. After 14 to 16 months of treatment, statistical analysis (analysis of variance) of data disclosed no significant differences in the overall performance of the two groups. A longer-duration trial, involving younger patients, is needed to determine whether there are palliative effects of penicillamine or other potentially therapeutic agents.