Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 studyNuria Carrillo, William A. Gahl, Galen O. Joe et al.|Genetics in Medicine|2021Cited by 55
Bayesian model of disease progression in GNE myopathyMelanie Quintana, Nuria Carrillo, Joseph A. Shrader et al.|Statistics in Medicine|2018Cited by 25