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3. Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis. Hastings C; Vieira C; Liu B; Bascon C; Gao C; Wang RY; Casey A; Hrynkow S Orphanet J Rare Dis; 2019 Oct; 14(1):228. PubMed ID: 31639011 [TBL] [Abstract][Full Text] [Related]
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