Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. [electronic resource]
Producer: 20071019Description: 765-72 p. digitalISSN:- 1474-4422
- 1-Deoxynojirimycin -- analogs & derivatives
- Adolescent
- Adult
- Age Factors
- Analysis of Variance
- Child
- Child, Preschool
- Cohort Studies
- Deglutition -- drug effects
- Double-Blind Method
- Drug Administration Schedule
- Enzyme Inhibitors -- therapeutic use
- Female
- Humans
- Male
- Niemann-Pick Disease, Type C -- drug therapy
- Retrospective Studies
- Saccades -- drug effects
- Severity of Illness Index
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Publication Type: Clinical Trial; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
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