Favorable outcomes following early onset oral miglustat in early infantile Niemann Pick Type C

Publication date: June 2021Source: Molecular Genetics and Metabolism Reports, Volume 27Author(s): Shiri Curelaru, Yoav Zehavi, Tal Almagor, Ronen Spiegel
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research