Abstract
This paper presents the rationale and results of our work on the use of penicillamine in the treatment of hereditary avian muscular dystrophy. We have shown that penicillamine, a cysteine analogue with a reduced sulfhydryl group, delays the onset of symptoms and alleviates the debilitating aspects of the disease (1). In avian dystrophy, deterioration of the muscle fibers is evidenced in the 2nd month by an inability of the birds to rise after falling on their backs and by a progressive rigidity of the wings. Penicillamine produced three major improvements: (a) better righting ability when birds were placed on their backs; (b) greater wing flexibility; (c) and suppression of plasma creatine phosphokinase activity. The beneficial effects of penicillamine on muscle function and biochemistry are discussed in terms of the mechanism of drug action.
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