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- 2019
Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart reviewDOI: 10.1186/s13023-019-0996-6 Keywords: Niemann-pick disease type C, NP-C, Disease characteristics, Prognosis, NP-C disability scales Abstract: The online version of this article (10.1186/s13023-019-0996-6) contains supplementary material, which is available to authorized users
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