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Facts About Metabolic Diseases of Muscle
Muscular Dystrophy Association |
Updated 04/03 |
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MDA's SEARCH FOR A TREATMENT OR CURE
MDA-funded scientists are pursuing a number of promising leads in their quest to understand the causes of the metabolic diseases of muscle.
To date, scientists have isolated all of the genes involved in the metabolic myopathies described in this brochure, and their genetic codes have been unraveled, offering insight into how particular gene defects lead to disease. In addition, isolation of genes has allowed researchers to begin experiments with gene therapy, a potential cure for some metabolic diseases.
The knowledge MDA-funded researchers are obtaining about the mechanisms by which metabolic gene defects cause disease may lead to other strategies for prevention and treatment. MDA's extensive research program includes studies to help in developing new treatments for metabolic diseases of muscle, such as strategies designed to replace the enzymes that are missing in these disorders.
| MDA's Web site is constantly updated with the latest information about the neuromuscular diseases in its program. See the latest research news. |
Targets now being pursued in research include:
- better diagnosis of metabolic diseases, to allow better identification of at-risk individuals and earlier treatment
- continued examination of the benefit of exercise in metabolic disease
- development of animal models of metabolic diseases, both to improve understanding of the diseases and to test possible treatments
- development of enzyme replacement therapy for acid maltase deficiency
- development of gene therapy for acid maltase deficiency and phosphorylase deficiency
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Facts About Metabolic Diseases of Muscle
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