Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study

Genetics in Medicine, Published online: 13 July 2021; doi:10.1038/s41436-021-01259-xSafety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study

Source: Genetics in Medicine - July 13, 2021 Category: Genetics & Stem Cells Authors: Nuria Carrillo May C. Malicdan Petcharat Leoyklang Joseph A. Shrader Galen Joe Christina Slota John Perreault John D. Heiss Bradley Class Chia-Ying Liu Kennan Bradley Colleen Jodarski Carla Ciccone Claire Driscoll Rebecca Parks Scott Van Wart Levent Bayma Source Type: research