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Publications
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. Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study. Genet Med. 2021 Nov;23(11):2067-75. doi: 10.1038/s41436-021-01259-x
Lewis MA, Stine A, Paquin RS, Mansfield C, Wood D, Rini C, Roche MI, Powell CM, Berg JS, Bailey, Jr DB. Parental preferences toward genomic sequencing for non-medically actionable conditions in children: a discrete-choice experiment. Genet Med. 2018 Feb;20(2):181-9. doi: 10.1038/gim.2017.93
Pavel M, Horsch D, Anthony L, Ervin C, Kulke MH, Bergsland E, Caplin M, Oberg K, Warner R, Kunz P, Metz DC, Pasieka J, Pavlakis N, DiBenedetti D, Lapuerta P. Patient interviews in TELESTAR, a phase 3 study of telotristat etiprate, report meaningful improvement in carcinoid syndrome. Poster presented at the 2016 European Neuroendocrine Tumor Society (ENETS) Annual Meeting; March 2016. Barcelona, Spain. [abstract] Neuroendocrinology. 2016; 103:89.