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 d...
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| Format: | Article |
| Language: | English |
| Published: |
Dove Medical Press
2009-11-01
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| Series: | Therapeutics and Clinical Risk Management |
| Online Access: | http://www.dovepress.com/new-therapies-in-the-management-of-niemann-pick-type-c-disease-clinica-a3687 |