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

Publication date: September 2021Source: Molecular Genetics and Metabolism Reports, Volume 28Author(s): Margaret Reynolds, Laura A. Linneman, Sofia Luna, Barbara B. Warner, Yumirle P. Turmelle, Sakil S. Kulkarni, Xuntian Jiang, Geetika Khanna, Marwan Shinawi, Forbes D. Porter, Daniel S. Ory, F. Sessions Cole, Patricia I. Dickson
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research