TREATMENT OF DUCHENNES MUSCULAR-DYSTROPHY WITH PENICILLAMINE - RESULTS OF A DOUBLE-BLIND TRIAL

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. © 1979, American Medical Association. All rights reserved.