Keens T, Hoffman V, Topuria I, Elder K, Cerf S, Mulder K, Roberts J, Lysinger J, Del Carmen Reyes M, Berdella M, Cairns AM, Jain M, Ganapathy V, Lou Y, Morcos B, Wu C, Sass L, VX19-CFD-003 Study Group. Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor on the burden of illness in adolescents and adults with cystic fibrosis. Heliyon. 2024 Mar 26;10(7):e28508. doi: 10.1016/j.heliyon.2024.e28508
Camprodon-Rosanas E, Pujol J, Martínez-Vilavella G, Blanco-Hinojo L, Medrano-Martorell S, Batlle S, Forns J, Ribas-Fito N, Dolz M, Sunyer J. Brain structure and function in school-aged children with sluggish cognitive tempo symptoms. J Am Acad Child Adolesc Psychiatry. 2019 Feb;58(2):256-96. doi: 10.1016/j.jaac.2018.09.441
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