Andrews S, Mansfield C, O'Neill C, Peay H. Meaningful treatment outcomes for Sanfilippo syndrome: using a combination of focus groups and best-worst scaling to elicit and explore parents' priorities. Poster presented at the 2019 25th Annual Qualitative Health Research Conference; October 2019. Vancouver, Canada. [abstract] Int J Qual Methods. 2020 Mar 30; 19:2. doi: 10.1177/1609406920909934
Vekeman F, Gauthier-Loiselle M, Faust E, Lefebvre P, Lahoz R, Duh MS, Sacco P. Patient and caregiver burden associated with fragile x syndrome in the United States. Am J Intellect Dev Disabil. 2015 Sep;120(5):444-59. doi: 10.1352/1944-7558-120.5.444