Title: | Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study |
Journal : | Genetics in medicine : official journal of the American College of Medical Genetics |
Authors: | Carrillo 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 ; Gahl WA |
Material Type: | Article |
Publisher: | United States, 13/07/2021 |
Languages: | English |
Keywords : | adulthood ; clinical trials ; GNE gene ; GNE myopathy ; N-acetylmannosamine (ManNac) |
Pubmed / DOI : | Pubmed : 34257421 / DOI : 10.1038/s41436-021-01259-x |
N° Profil MNM : | 2021071 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/34257421 |
See also : |