Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Barrett AM, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: long-term safety and efficacy results of the 32-month, open-label LILAC-2 study. Med. 2024 Oct 11;5(10):1275-1281. doi: 10.1016/j.medj.2024.06.007
McCann E, Peterson KA, Whalley D, Qin S, Tilton ST, Kamat S, Sun X, Dellon ES. Psychometric evaluation of the Dysphagia Symptom Questionnaire for adults and adolescents with eosinophilic esophagitis. J Allergy Clin Immunol Glob. 2024 Jul 16;3(4):100302. doi: 10.1016/j.jacig.2024.100302
Raspa M, Sacco P, Candrilli SD, Bishop E, Petrillo J. Validity of a condition specific outcome measure for fragile X syndrome: the Aberrant Behaviour Checklist-utility index. J Intellect Disabil Res. 2016 Sep;60(9):844-55. doi: 10.1111/jir.12264