TY  - GEN
AU  - Patterson,Marc C
AU  - Vecchio,Darleen
AU  - Prady,Helena
AU  - Abel,Larry
AU  - Wraith,James E
TI  - Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
SN  - 1474-4422
PY  - 2007///1019
KW  - 1-Deoxynojirimycin
KW  - analogs & derivatives
KW  - Adolescent
KW  - Adult
KW  - Age Factors
KW  - Analysis of Variance
KW  - Child
KW  - Child, Preschool
KW  - Cohort Studies
KW  - Deglutition
KW  - drug effects
KW  - Double-Blind Method
KW  - Drug Administration Schedule
KW  - Enzyme Inhibitors
KW  - therapeutic use
KW  - Female
KW  - Humans
KW  - Male
KW  - Niemann-Pick Disease, Type C
KW  - drug therapy
KW  - Retrospective Studies
KW  - Saccades
KW  - Severity of Illness Index
N1  - Publication Type: Clinical Trial; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1016/S1474-4422(07)70194-1
ER  -