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Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)
BACKGROUND: Mucopolysaccharidosis (MPS) type III (Sanfilippo syndrome) comprises a group of rare, lysosomal storage diseases caused by the deficiency of one of four enzymes involved in the degradation of heparan sulfate. The clinical hallmark of the disease is severe neurological deterioration leadi...
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| Publicado no: | Orphanet J Rare Dis |
|---|---|
| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6498678/ https://ncbi.nlm.nih.gov/pubmed/31046785 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-019-1065-x |
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