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Phase 1/2a trial of intrathecal 2-hydroxypropyl-beta-cyclodextrin (VTS-270) for the treatment of Niemann-Pick Disease, type C1

Document Type

Journal

Author

Porter, F. D.; Farhat, N. Y.; Ottinger, E.; Mckew, J. C.; Weissfeld, L.; Machielse, B. N.; Berry-Kravis, E. M.; Vite, H.; Ory, D. S.; Bianconi, S.; TRND Team

Source

EUROPEAN JOURNAL OF HUMAN GENETICS; OCT 2018, 26 p405-p406, 2p. Supplement: S

Subject

Language

English

ISSN

14765438

Online Access

Full Text (ProQuest Central) Web of Science JCR 저널정보 Find it@PNU

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