Muscle Nerve. 2007 Jan;35(1):70-7.

Modification of the natural history of adult-onset acid maltase deficiency by nutrition and exercise therapy.

Slonim AE, Bulone L, Goldberg T, Minikes J, Slonim E, Galanko J, Martiniuk F.

Source

Department of Pediatrics, Division of Molecular Genetics, Columbia University Medical College, 3959 Broadway BH6N, Room 601A, New York, New York 10032, USA. as2718@columbia.edu

Abstract

Adult-onset acid maltase deficiency is an inherited lysosomal skeletal-muscle disease characterized by progressive myopathy and respiratory failure, for which there is no known therapy. In an uncontrolled, prospective study, we evaluated whether adherence to high-protein and low-carbohydrate nutrition and exercise therapy (NET) can slow the progressive deterioration of muscle function in this disease. Thirty-four patients have been treated with NET for periods of 2-10 years (mean 4.5 +/- 2.5). Pre-NET rate of muscle function deterioration, as measured by the Walton scale, was compared to post-NET rate. Twenty-six patients were deemed to be consistently compliant with NET. Difference between pre-NET slope of muscle function deterioration to that of post-NET slope in compliant patients was -0.29 (95% CI -0.19, 0.39) (P < 0.0001). We conclude that compliance with NET can slow deterioration of muscle function and improve the natural history of adult-onset acid maltase deficiency. Muscle Nerve, 2006.

PMID:: 17022069; [PubMed - indexed for MEDLINE]

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