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Therapies of mucopolysaccharidosis IVA (Morquio A syndrome)
INTRODUCTION: Morquio A syndrome (mucopolysaccharidosis type IVA, MPS IVA) is one of the lysosomal storage diseases and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme leads to accumulation of glycosaminoglycans (GAGs), keratan sulfate (KS)...
Shranjeno v:
| izdano v: | Expert Opin Orphan Drugs |
|---|---|
| Main Authors: | , , , , , , , , , , , |
| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
2013
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| Teme: | |
| Online dostop: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4238063/ https://ncbi.nlm.nih.gov/pubmed/25419501 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2013.846853 |
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