Results of 14-year-long Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II: Clinical Case
Background. Mucopolysaccharidosis type II (MPS II) is a rare hereditary disease from the group of lysosomal storage diseases, with progressive course. There is effective enzyme replacement therapy (ERT) for this disease, it prevents the development of severe complications and improves patients'...
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| Main Authors: | , , , , , , , |
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| Format: | Book |
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"Paediatrician" Publishers LLC,
2023-01-01T00:00:00Z.
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