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Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I
BACKGROUND: Mucopolysaccharidosis type I can be classified as three clinical sub‐types; Hurler syndrome, Hurler‐Scheie syndrome and Scheie syndrome, with the scale of severity being such that Hurler syndrome is the most severe and Scheie syndrome the least severe. It is a rare, autosomal recessive d...
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| Izdano u: | Cochrane Database Syst Rev |
|---|---|
| Glavni autori: | , , |
| Format: | Artigo |
| Jezik: | Inglês |
| Izdano: |
John Wiley & Sons, Ltd
2019
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| Online pristup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6581069/ https://ncbi.nlm.nih.gov/pubmed/31211405 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD009354.pub5 |
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