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

PURPOSE: To evaluate the safety and efficacy of N-acetylmannosamine (ManNAc) in GNE myopathy, a genetic muscle disease caused by deficiency of the rate-limiting enzyme in N-acetylneuraminic acid (Neu5Ac) biosynthesis. METHODS: We conducted an open-label, phase 2, single-center (NIH, USA) study to ev...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8553608/
https://www.ncbi.nlm.nih.gov/pubmed/34257421
http://dx.doi.org/10.1038/s41436-021-01259-x

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