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May 1983

Effects of Allopurinol in Duchenne's Muscular Dystrophy

Author Affiliations

From the Departments of Medicine (Drs Hunter, Galloway, and Rudman, and Ms Gerron), Rehabilitation (Dr Brooke and Ms Wardlaw), and Biometry (Dr Kutner and Mr Vogel), Clinical Research Facility, Emory University School of Medicine, Atlanta.

Arch Neurol. 1983;40(5):294-299. doi:10.1001/archneur.1983.04050050062009

• There is no effective treatment at present for Duchenne's muscular dystorphy (DMD). Recently, clinical improvement and increased muscle strength have been reported after 100 mg of allopurinol was administered daily for 41/2 months to 16 boys with DMD. Our objective was to validate the effectiveness of 100 mg of allopurinol given daily to 21 boys with DMD in a double-blind crossover study. Its effectiveness was determined by an extensive battery of muscle function, cardiac, and laboratory tests. The ten-month study was divided into four periods: (1) one month of baseline observation; (2) three months of therapy to allopurinol and placebo control groups; (3) three months of therapy to crossed over groups; and (4) three months of therapy to all patients. No statistically significant changes between the allopurinol and control periods occurred in the muscle function tests. The earlier report of clinical improvement during allopurinol treatment was not confirmed.

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