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 Date: 2013-07-21 06:20:11Rare diseases Medicine Niemann–Pick disease Niemann-Pick disease type C Cyclodextrin Lysosomal storage disease European Medicines Agency Miglustat Orphan drug Health Chemistry Lipid storage disorders |
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| -Application for Renewal of RDMM Catalyst Grants This application is to be used by the Network’s Scientific Advisory Committee (SAC) to determine as to whether to provide an additional year of funding ($DocID: 1vceh - View Document |
| MULTISYSTEMIC RARE DISEASESDocID: 1v3AN - View Document |