TY  - GEN
AU  - Patterson,Marc C
AU  - Mengel,Eugen
AU  - Vanier,Marie T
AU  - Schwierin,Barbara
AU  - Muller,Audrey
AU  - Cornelisse,Peter
AU  - Pineda,Mercè
TI  - Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study
SN  - 1750-1172
PY  - 2016///0229
KW  - 1-Deoxynojirimycin
KW  - analogs & derivatives
KW  - Adolescent
KW  - Adult
KW  - Child
KW  - Child, Preschool
KW  - Cohort Studies
KW  - Disease Progression
KW  - Enzyme Inhibitors
KW  - therapeutic use
KW  - Female
KW  - Humans
KW  - Male
KW  - Niemann-Pick Disease, Type C
KW  - drug therapy
KW  - Prospective Studies
KW  - Treatment Outcome
KW  - Young Adult
N1  - Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1186/s13023-015-0284-z
ER  -