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Observing the advanced disease course in mucopolysaccharidosis, type IIIA; a case series.
This follow-up study of a subgroup of the patients seen in a natural history study of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A) addressed the adaptive and medical characteristics of their advanced disease manifestations. Of the original 24 patients, specific data was collected on...
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| Publicado no: | Mol Genet Metab |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7814869/ https://ncbi.nlm.nih.gov/pubmed/29198891 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.11.014 |
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