Clinical investigation in Duchenne muscular dystrophy: Penicillamine and vitamin E

Gerald M. Fenichel, Michael H. Brooke, Robert C. Griggs, Jerry R. Mendell, J. Phillip Miller, Richard T. Moxley, Jane H. Park, Michael A. Provine, Julaine Florence, Kenneth K. Kaiser, Wendy M. King, Shree Pandya, Jenny Robison, Linda Signore

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A double‐blind controlled trial of penicillamine and vitamin E against placebo was conducted in 106 boys with Duchenne muscular dystrophy. Nine dropped out of the study after being randomized. Patients were evaluated using an established protocol that measures muscle strength, joint contractures, functional grade, and pulmonary function. The trial lasted for 18 months. The power of the study to detect a 75% slowing of the disease was 0.95 at a P < 0.05. No therapeutic effect was observed in the patients taking penicillamine and vitamin E as compared with those taking placebo.

Original languageEnglish
Pages (from-to)1164-1168
Number of pages5
JournalMuscle & Nerve
Issue number11
StatePublished - Nov 1988


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