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

por Carrillo, Nuria, Malicdan, May C., Leoyklang, Petcharat, Shrader, Joseph A., Joe, Galen, Slota, Christina, Perreault, John, Heiss, John D., Class, Bradley, Liu, Chia-Ying, Bradley, Kennan, Jodarski, Colleen, Ciccone, Carla, Driscoll, Claire, Parks, Rebecca, Van Wart, Scott, Bayman, Levent, Coffey, Christopher S., Quintana, Melanie, Berry, Scott M., Huizing, Marjan, Gahl, William A.
Publicado 2021