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Impact of Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin (HP-B-CD, VTS-270) on Cognitive Decline in Niemann-Pick Type C1 (NPC1)

E. Berry-Kravis,J. Chin, R. Jaeger, K. Friedmann,A. Hoffmann,A. Wainer,C. Leonczyk, L. Soorya,A. Thurm,C. Farmer, N. Farhat, F. Porter

ANNALS OF NEUROLOGY(2019)

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关键词
Rare Diseases,Translational/Experimental Therapeutics,Genetics
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