Chronic allopurinol and adenine therapy in Duchenne muscular dystrophy

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Abstract
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.