A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-β-cyclodextrin for acute liver disease in infants with Niemann-Pick C1
Mol Genet Metab Rep
Margaret Reynolds 1, Laura A Linneman 1, Sofia Luna 1, Barbara B Warner 1, Yumirle P Turmelle 1, Sakil S Kulkarni 1, Xuntian Jiang 1, Geetika Khanna 1, Marwan Shinawi 1, Forbes D Porter 2, Daniel S Ory 1, F Sessions Cole 1, Patricia I Dickson 1
DOI: 10.1016/j.ymgmr.2021.100772
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