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Disorders resulting from an inherited deficiency or defect of a single enzyme or protein.
They include the mucopolysaccharidoses (MPS), the leukodystrophies such as Adrenoleukodystrophy (ALD), Metachromatic Leukodystrophy (MLD), and Globoid Leukodystrophy (GLD), and other storage diseases, such as alpha-mannosidosis, fucosidosis, and I-cell disease.
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