Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures
Mucopolysaccharidosis VI (MPS VI) is an inheritable, clinically heterogeneous lysosomal storage disorder that develops due to a deficiency in the arylsulfatase B (ASB) enzyme. This deficiency impairs the stepwise degradation of glycosaminoglycans (GAGs) resulting in the accumulation of partia...
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| Format: | Article |
| Language: | English |
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Hacettepe University Institute of Child Health
2010-10-01
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| Series: | The Turkish Journal of Pediatrics |
| Online Access: | https://turkjpediatr.org/article/view/5045 |