Safety and Efficacy of N-Acetylmannosamine (ManNAc) in Patients with GNE Myopathy: An Open-Label Phase 2 Study

Carillo N, Malicdan MC, Leoyklang P, Shrader JA, Joe G, Slota C, Perreault J, Heiss JD, Class B, Liu CY, Bradley K, Jodarski C, Ciccone C, Driscoll C, Parks R, Van Wart S, Bayman L, Coffey CS, Quintana M, Berry SM, Huizing M, and Gahl WA