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NEUROLOGY 1985;35:61
© 1985 American Academy of Neurology

Chronic allopurinol and adenine therapy in Duchenne muscular dystrophy

Effects on muscle function, nucleotide degradation, and muscle ATP and ADP content

T. E. Bertorini, G.M.A. Palmieri, J. Griffin, C. Chesney, D. Pifer, L. Verling, D. Airozo and I. H. Fox

Departments of Neurology, Medicine, and Pathology, University of Tennessee Center for the Health Sciences, Memphis, TN, and the Human Purine Research Center, Departments of Internal Medicine and Biological Chemistry, University of Michigan, Ann Arbor.

Prompted by the controvery on the efficacy of allopurinol in Duchenne muscular dystrophy and by our observations of an abnormal adenine nucleotide turnover in this disease, we conducted an 18 month, double-blind clinical trial with allopurinol and adenine in 14 Duchenne boys paired according to age and functional activity. Detailed clinical evaluation was performed trimonthly. Muscle ATP and ADP content was measured before and ahr 1 year of treatment. The effect of therapy on adenine nucleotide turnover was determined. No significant difference was observed between the treated and placebo groups, but both showed a significant deterioration (p < 0.05) in most clinical parameters. Muscle ATP was reduced in Duchenne dystrophy (p < 0.02) but did not change with therapy, and no correction of the abnormal adenine nucleotide degradation was observed.

Address correspondence and reprint requests to Dr. Bertorini, University of Tennessee Center for the Health Sciences, Department of Neurology, 956 Court Avenue, Room 2B03, Memphis, TN 38163.

Supported by a grant from the Muscular Dystrophy Association, Inc., and by USPHS grants RR002211, 5-M01 RR42 and AM-19674.

Accepted for publication April 4, 1984.




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