Skip to main content
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
Boccia S, Rothman KJ, Panic N, Flacco ME, Rosso A, Pastorino R, Manzoli L, La Vecchia C, Villari P, Boffetta P, Ricciardi W, Ioannidis JP. Registration practices for observational studies on ClinicalTrials.gov indicated low adherence. J Clin Epidemiol. 2016 Feb;70:176-82. doi: 10.1016/j.jclinepi.2015.09.009