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Pharmacological and nutritional treatment trials in McArdle disease
1The Wolfson Centre for Inherited Neuromuscular Diseases (CIND), Robert Jones and Agnes Hunt, NHS Trust, Oswestry, UK. Rcmq37@aol.com
Abstract:
A systematic review of evidence for randomised controlled trials using pharmacologic and nutritional therapies in McArdle disease was undertaken. Primary outcome measures included any objective assessment of exercise endurance. Secondary outcome measures included changes in metabolic parameters, subjective measures such as quality of life scores and adverse outcomes. Ten randomised controlled trials were identified. Two trials low dose creatine (60 mg/kg/day) and oral sucrose 75 g prior to exercise demonstrated a positive effect.
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