GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS
Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured. However, with the arrival of effective gene therapy, to...
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| Main Authors: | , , , , , , , |
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| Format: | Book |
| Published: |
The Society for Children with Metabolic Disorders,
2022-05-01T00:00:00Z.
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