Articles

Search Articles

Advanced search
Help

Catalogue

Articles

Databases

X
Search Filters
Format Format
Subjects Subjects
Subjects Subjects
X
Sort by Item Count (A-Z)
Filter by Count
1-deoxynojirimycin (1) 1
1-deoxynojirimycin - analogs & derivatives (1) 1
1-deoxynojirimycin - therapeutic use (1) 1
610 medicine & health (1) 1
adolescent (1) 1
adult (1) 1
analysis (1) 1
care and treatment (1) 1
child (1) 1
child, preschool (1) 1
children (1) 1
clinical medicine (1) 1
cohort studies (1) 1
development and progression (1) 1
disease progression (1) 1
drug therapy (1) 1
dysphagia (1) 1
enzyme inhibitors (1) 1
enzyme inhibitors - therapeutic use (1) 1
female (1) 1
genetics (1) 1
genetics & heredity (1) 1
humans (1) 1
index medicus (1) 1
journal article (1) 1
klinisk medicin (1) 1
male (1) 1
medical and health sciences (1) 1
medical clinic (1) 1
medicin och hälsovetenskap (1) 1
medicine (1) 1
medicine, research & experimental (1) 1
miglustat (1) 1
nervous system diseases (1) 1
neurologi (1) 1
neurology (1) 1
niemann-pick disease (1) 1
niemann-pick disease type c (1) 1
niemann-pick disease, type c (1) 1
niemann-pick disease, type c - drug therapy (1) 1
niemann-pick disease, type c - pathology (1) 1
pediatric-patients (1) 1
pharmacology (1) 1
pick's disease (1) 1
prospective studies (1) 1
registry (1) 1
research (1) 1
research support, n.i.h., extramural (1) 1
research support, non-u.s. gov't (1) 1
treatment outcome (1) 1
young adult (1) 1
more...
Language Language
Publication Date Publication Date
Click on a bar to filter by decade
Slide to change publication date range


1. Full Text 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
by Patterson, Marc C and Mengel, Eugen and Vanier, Marie T and Schwierin, Barbara and Muller, Audrey and Cornelisse, Peter and Pineda, Mercè and Amado-Fondo, A and Amraoui, Y and Andria, G and Arellano, M and Audoin, B and Azcona, C and Barr, C and Baruteau, J and Baumgartner, C and Bell, L and Bembi, B and Benneddif, K and Bernard, G and Bobocea, N and Bodzioch, M and Boettcher, T and Bonnan, M and Broue, P and Bruni, A and Caceres, M and Camino, R and Campbell, E and Cances, C and Cannell, P and Cesar, J and Chabrol, B and Chakrapani, A and Colao, R and Collet, A and Corsetti, T and Cousins, A and Covanis, A and Cox, T and Cuisset, J.M and Dardis, A and Das, A and Deegan, P and Dengler, T and Deodato, F and Derralynn, H and Di Donato, I and Di Rocco, M and Dinopoulos, A and Pakiela, Domanska and Eckehard, S and Engelen, M and Eyer, D and Fecarotta, S and Federico, A and Filla, A and Fiumara, A and Fonseca, M.J and Gabrielli, O and Garcia, T and Garrote, J and Gissen, P and Giugliani, L and Greenberg, C and Heron, B and Hertzberg, C and Higgins, F and Hill, A and Hiwot, T and Hlavata, A and Hörbe-Blindt, A and Howley, E and Hussain, N and Illsinger, S and Imrie, J and Jacklin, E and Jones, S and Jovanovic, A and Kaczmarek, V and Kaphan, E and Kibaek, M and Kleinhans, P and Klünemann, K.H and Koch, S.M and Koegl-Wallner, W and Kolnikova, M and Korenke, G.C and Korinthenberg, R and Kumari, S and Lachmann, R and Lee Ann, L and Likopoulou, L and Lilienthal, E and Link, B and Lippold, M and Lopez-Laso, E and Luecke, T and Lundgren, J and Mackrell, M and ... and NPC Registry Investigators and NPC Registry investigators and On behalf of the NPC Registry investigators and Child and Adolescent Psychiatry and Lund University and Klinisk neurogenetik and Barn- och ungdomspsykiatri and MultiPark: Multidisciplinary research focused on Parkinson´s disease and Clinical Neurogenetics and Lunds universitet
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 05/2015, Volume 10, Issue 1, p. 65
Background: Niemann-Pick disease type C (NP-C) is a rare neurovisceral disease characterised by progressive neurological degeneration, where the rate of... 
Miglustat | Niemann-Pick disease type C | Registry | DYSPHAGIA | MEDICINE, RESEARCH & EXPERIMENTAL | PEDIATRIC-PATIENTS | GENETICS & HEREDITY | CHILDREN | Niemann-Pick Disease, Type C - drug therapy | Prospective Studies | Humans | Child, Preschool | Male | Treatment Outcome | Enzyme Inhibitors - therapeutic use | Niemann-Pick Disease, Type C - pathology | Disease Progression | Young Adult | 1-Deoxynojirimycin - therapeutic use | Adolescent | 1-Deoxynojirimycin - analogs & derivatives | Adult | Female | Child | Cohort Studies | Niemann-Pick disease | Development and progression | Research | Drug therapy | Pick's disease | Care and treatment | Nervous system diseases | Analysis | Neurologi | Enzyme Inhibitors | Medical and Health Sciences | Medicin och hälsovetenskap | 1-Deoxynojirimycin | Klinisk medicin | Journal Article | Research Support, N.I.H., Extramural | Clinical Medicine | Neurology | Research Support, Non-U.S. Gov't | Niemann-Pick Disease, Type C
Journal Article
Details down arrow
Digital rights down arrow
loading Loading Rights
No results were found for your search.

Cannot display more than 1000 results, please narrow the terms of your search.