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3. Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin. Berry-Kravis E; Chin J; Hoffmann A; Winston A; Stoner R; LaGorio L; Friedmann K; Hernandez M; Ory DS; Porter FD; O'Keefe JA Pediatr Neurol; 2018 Mar; 80():24-34. PubMed ID: 29429782 [TBL] [Abstract][Full Text] [Related]
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