New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
James E Wraith, Jackie ImrieWillink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, Manchester, UKAbstract: Niemann-Pick disease type C (NP-C) is an autosomal recessive disorder characterized by progressive neurological deterioration leading to premature death. Th...
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| Formato: | Livro |
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Dove Medical Press,
2009-11-01T00:00:00Z.
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