[Report of attendance at the Parseghian Scientific Conference for Niemann-Pick Type C (NP-C) Research, South Bend, Indiana: in search of new therapeutic possibilities for a very rare illness].
beck_fornfeist@gmx.de
Sabine
S
Fornfeist
D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives / Q000627:therapeutic use; D000073738:2-Hydroxypropyl-beta-cyclodextrin; D000293:Adolescent; D000818:Animals; D002648:Child; D004195:Disease Models, Animal; D004200:Diseases in Twins; D015507:Drugs, Investigational / Q000627:therapeutic use; D006655:Histone Deacetylases / Q000627:therapeutic use; D006801:Humans; D007262:Infusions, Intravenous; D007278:Injections, Spinal; D051379:Mice; D052556:Niemann-Pick Disease, Type C / Q000175:diagnosis / Q000188:drug therapy / Q000451:nursing*; D012106:Research; D014481:United States; D014486:United States Food and Drug Administration; D047392:beta-Cyclodextrins / Q000627:therapeutic use
D015507:Drugs, Investigational; D047392:beta-Cyclodextrins; D017485:1-Deoxynojirimycin; D000073738:2-Hydroxypropyl-beta-cyclodextrin; C059896:miglustat; D006655:Histone Deacetylases